Archive for October, 2022

NAD+ Cell Regenerator and Resveratrol Elite combines NIAGEN nicotinamide riboside and ultra-bioavailable forms resveratrol with quercetin and fisetin to create an innovative nutritional supplement for longevity and youthful cellular energy production.

Resveratrol is a well-known longevity and anti-aging supplement.1 Our Resveratrol Elite formulas contains trans-resveratrol, the form associated with beneficial biological effects.2-5 Resveratrol also promotes healthy insulin sensitivity, supports a healthy inflammatory response and has been shown to promote healthy endothelial function for a healthy cardiovascular system.4,6,7

A more bioavailable resveratrol

Weve combined resveratrol with galactomannan fibers from fenugreek seeds. This makes our Resveratrol Elite formulas up to 10 times more bioavailable. This means that the trans-resveratrol in our supplements reaches higher levels in your bloodstream and circulates longer than traditional, unformulated resveratrol.

Resveratrol and heart health

One way that resveratrol promotes heart health is by helping to shield the body from oxidative stress at the cellular level.8 By inhibiting oxidative stress in your cardiovascular system, resveratrol promotes endothelial healthan essential component of living a long, healthy life.9

Resveratrol and brain health

At the same time, resveratrols ability to support cerebrovascular blood flow may also make it good for your brain: there is clinical evidence that resveratrol can help encourage youthful neurological function and with it, things like cognition. Interestingly, this same trial showed that resveratrol promoted healthy glucose metabolismanother essential aspect of healthy longevity.10

Fight general fatigue with NIAGEN

Nicotinamide riboside increases your bodys levels of NAD+, a coenzyme critical to healthy cellular function.11 In a randomized controlled trial published in 2017, older adults taking a daily dose of 250 mg of nicotinamide riboside had a 40% increase in NAD+ levels after just 30 days.2 Studies in preclinical models have shown that increasing NAD+ also encouraged healthy metabolic and cognitive function.3,4

Fisetin and quercetin

Fisetin and quercetin are phytonutrient compounds that augment trans-resveratrols healthy effects, promote cardiovascular health, support healthy cellular function, fight oxidative stress, promote a healthy inflammatory response and more.9,10

Take the fight against aging to the cellular level with NAD+ Cell Regenerator and Resveratrol Elite.

Read more from the original source:
NAD+ Cell Regenerator and Resveratrol Elite - Life Extension

For many patients battling deadly diseases, getting access to a clinical trial can be life-saving, but it can also be very challenging. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved a concept plan to make it financially and logistically easier for patients to take part in CIRM-funded clinical trials.

The plan will create a Patient Support Program (PSP) to provide support to California patients being evaluated or enrolled in CIRM-supported clinical trials, with a particular emphasis on helping underserved populations.

Helping scientists develop stem cell and gene therapies is just part of what we do at CIRM. If those clinical trials and resulting therapies are not accessible to the people of California, who are making all this possible, then we have not fulfilled our mission. says Maria T. Millan, M.D., President and CEO of CIRM.

The Patient Support Plan will offer a range of services including:

The funds for the PSP are set aside under Proposition 14, the voter-approved initiative that re-funded CIRM in 2020. Under Prop 14 CIRM money that CIRM grantees earn from licensing, inventions or technologies is to be spent offsetting the costs of providing treatments and cures arising from institute-funded research to California patients who have insufficient means to purchase such treatment or cure, including the reimbursement of patient-qualified costs for research participants.

Currently, the CIRM Licensing Revenues and Royalties Fund has a balance of $15.6 million derived from royalty payments.

The patient support program and financial resources will not only help patients in need, it will also help increase the likelihood that these clinical trials will succeed, says Sean Turbeville, Ph.D., Vice President of Medical Affairs and Policy at CIRM. We know cell and gene therapies can be particularly challenging for patients and their families. The financial challenges, the long-distance traveling, extended evaluation, and family commitments can make it difficult to enroll and retain patients. The aim of the PSP is to change that.

The overall objective of this funding opportunity is to establish a statewide program that, over five years, is expected to support hundreds of patients in need as they participate in the growing number of CIRM-supported clinical trials. The program is expected to cost between $300,000 to $500,000 a year. That money will come from the Medical Affairs budget and not out of the patient assistance fund.

The first phase of the program will identify an organization, through a competitive process, that has the expertise to provide patient support services including:

You can find more information about the Patient Support Program on our website here and here.

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Continued here:
State Stem Cell & Gene Therapy Agency Sets up Support Program to Help ...

CRISPR is a technology that can be used to edit genes and, as such, will likely change the world.

The essence of CRISPR is simple: its a way of finding a specific bit of DNA inside a cell. After that,the next step in CRISPR gene editing is usually to alter that piece of DNA. However, CRISPR has also been adapted to do other things too, such as turning genes on or off without altering their sequence.

There were ways to edit the genomes of some plants and animals before the CRISPR method was unveiled in 2012 but it took years and cost hundreds of thousands of dollars. CRISPR has made it cheap and easy.

CRISPR is already widely used for scientific research, and in the not too distant future many ofthe plantsandanimalsinour farms, gardens or homes may have been altered with CRISPR. In fact, some people already are eating CRISPRed food.

CRISPR technology also has the potential to transform medicine, enabling us to not onlytreatbut alsopreventmany diseases. We may even decide to use it tochange the genomesofour children. An attempt to do this in Chinahasbeen condemned as premature and unethical, but some think it could benefit children in the future.

CRISPR is being used for all kinds of other purposes too, from fingerprinting cells andlogging what happensinside them todirecting evolutionand creatinggene drives.

The key to CRISPR is the many flavours of Cas proteins found in bacteria, where they help defend against viruses. The Cas9 protein is the most widely used by scientists. This protein can easily be programmed to find and bind to almost any desired target sequence, simply by giving it a piece of RNA to guide it in its search.

When the CRISPR Cas9 protein is added to a cell along with a piece of guide RNA, the Cas9 protein hooks up with the guide RNA and then moves along the strands of DNA until it finds and binds to a 20-DNA-letter long sequence that matches part of the guide RNA sequence. Thats impressive, given thatthe DNA packed into each of our cellshas six billion letters and is two metres long.

What happens next can vary. The standard Cas9 protein cuts the DNA at the target. When the cut is repaired, mutations are introduced that usually disable a gene. This is by far the most common use of CRISPR. Its called genome editing or gene editing but usually the results arenot as preciseas that term implies.

CRISPR can also be used tomake precise changessuch as replacing faulty genes true genome editing but this is far more difficult.

Customised Cas proteins have been created that do not cut DNA or alter it in any way,but merely turn genes on or off: CRISPRa and CRISPRi respectively. Yet others, called base editors,change one letter of the DNA code to another.

So why do we call it CRISPR? Cas proteins are used by bacteria to destroy viral DNA. They add bits of viral DNA to their own genome to guide the Cas proteins, and the odd patterns of these bits of DNA are what gave CRISPR its name: clustered regularly interspaced short palindromic repeats. Michael Le Page

Read more:
What is CRISPR? | New Scientist

Many bacterial clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated (Cas) systems employ the dual RNA-guided DNA endonuclease Cas9 to defend against invading phages and conjugative plasmids by introducing site-specific double-stranded breaks in target DNA. Target recognition strictly requires the presence of a short protospacer adjacent motif (PAM) flanking the target site, and subsequent R-loop formation and strand scission are driven by complementary base pairing between the guide RNA and target DNA, Cas9-DNA interactions, and associated conformational changes. The use of CRISPR-Cas9 as an RNA-programmable DNA targeting and editing platform is simplified by a synthetic single-guide RNA (sgRNA) mimicking the natural dual trans-activating CRISPR RNA (tracrRNA)-CRISPR RNA (crRNA) structure. This review aims to provide an in-depth mechanistic and structural understanding of Cas9-mediated RNA-guided DNA targeting and cleavage. Molecular insights from biochemical and structural studies provide a framework for rational engineering aimed at altering catalytic function, guide RNA specificity, and PAM requirements and reducing off-target activity for the development of Cas9-based therapies against genetic diseases.

Keywords: CRISPR; Cas9; genome engineering; mechanism; off-target; structure.

Read the original:
CRISPR-Cas9 Structures and Mechanisms - PubMed

In July, an HIV-positive man became the first volunteer in a clinical trial aimed at using Crispr gene editing to snip the AIDS-causing virus out of his cells. For an hour, he was hooked up to an IV bag that pumped the experimental treatment directly into his bloodstream. The one-time infusion is designed to carry the gene-editing tools to the mans infected cells to clear the virus.

Later this month, the volunteer will stop taking the antiretroviral drugs hes been on to keep the virus at undetectable levels. Then, investigators will wait 12 weeks to see if the virus rebounds. If not, theyll consider the experiment a success. What were trying to do is return the cell to a near-normal state, says Daniel Dornbusch, CEO of Excision BioTherapeutics, the San Francisco-based biotech company thats running the trial.

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Crispr isbeing used in several other studiesto treat a handful of conditions that arise from genetic mutations. In those cases, scientists are using Crispr to edit peoples own cells. But for the HIV trial, Excision researchers are turning the gene-editing tool against thevirus. The Crispr infusion contains gene-editing molecules that target two regions in the HIV genome important for viral replication. The virus can only reproduce if its fully intact, so Crispr disrupts that process by cutting out chunks of the genome.

This is an excerpt. Read the full article here

More here:
A CRISPR cure for HIV? Gene-editing technology may be able stop viral replication in its tracks and wipe out infections - Genetic Literacy Project

Studies in non-human primates demonstrated nearly 100% gene editing and knockout of endogenous RHO gene and more than 30% replacement protein levels using a dual vector AAV approach

Treated eyes showed morphological and functional photoreceptor preservation

EDIT-103 advancing towards IND-enabling studies

CAMBRIDGE, Mass., Oct. 13, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced ex vivo and in vivo preclinical data supporting its experimental medicine EDIT-103 for the treatment of rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP). The Company reported these data in an oral presentation today at the European Society of Gene and Cell Therapy 29th Annual Meeting in Edinburgh, Scotland, UK.

EDIT-103 is a mutation-independent CRISPR/Cas9-based, dual AAV5 vectors knockout and replace (KO&R) therapy to treat RHO-adRP. This approach has the potential to treat any of over 150 dominant gain-of-function rhodopsin mutations that cause RHO-adRP with a one-time subretinal administration.

These promising preclinical data demonstrate the potential of EDIT-103 to efficiently remove the defective RHO gene responsible for RHO-adRP while replacing it with an RHO gene capable of producing sufficient levels of RHO to preserve photoreceptor structure and functions. The program is progressing towards the clinic, said Mark S. Shearman, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. EDIT-103 uses a dual AAV gene editing approach, and also provides initial proof of concept for the treatment of other autosomal dominant disease indications where a gain of negative function needs to be corrected.

Key findings include:

Full details of the Editas Medicine presentations can be accessed in the Posters & Presentations section on the Companys website.

About EDIT-103EDIT-103 is a CRISPR/Cas9-based experimental medicine in preclinical development for the treatment of rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), a progressive form of retinal degeneration. EDIT-103 is administered via subretinal injection and uses two adeno-associated virus (AAV) vectors to knockout and replace mutations in the rhodopsin gene to preserve photoreceptor function. This approach can potentially address more than 150 gene mutations that cause RHO-adRP.

AboutEditas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Harvard and Broad Institutes Cas9 patent estates and Broad Institutes Cas12a patent estate for human medicines. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "target," "should," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Annual Report on Form 10-K, which is on file with theSecurities and Exchange Commission, as updated by the Companys subsequent filings with theSecurities and Exchange Commission, and in other filings that the Company may make with theSecurities and Exchange Commissionin the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

Original post:
Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the European Society of Gene...

Gene editing has long been primarily used for research, treatment, and disease prevention. Currently, this technology is increasingly being applied to modify agricultural products to create more perfect species. More and more genetically edited foods are appearing on the market, including high-nutrient tomatoes and zero-trans-fat soybean oil.

Some argue that gene-edited foods are safer than genetically modified (GM) foods (pdf). The U.S. Department of Agriculture (USDA) specified in 2018 that most genetically edited foods do not need to be regulated. However, are these foods, which will increasingly appear on the table, really risk-free?

In September 2021, the first gene-edited foodSicilian Rouge tomatoesmade with CRISPR-Cas9 technology were officially on sale.

This gene-edited tomato contains high levels of gamma-aminobutyric acid (GABA), which helps lower blood pressure and aids relaxation.

Japanese researchers remove a gene from the genome of the common tomato. After the gene is removed, the activity of an enzyme in tomatoes increases, promoting the production of GABA. The GABA content in this tomato is four to five times higher than that of a regular tomato.

Warren H. J. Kuo, an emeritus professor of the Department of Agronomy at National Taiwan University, explains that both gene editing and transgenic organisms are genetic modification, also known as genetic engineering.

The earliest technique was genetic modification, that is, transgenicin which a plant or animal is being inserted a gene from another species, such as a specific bacterial gene. The purpose of artificially modifying plants and animals is to improve their resistance against diseases and droughts, promote growth rates, increase yields, or improve nutrient content. However, the finished product will exhibit the foreign species genes.

Kuo says that transgenic modification is genetic modification 1.0, while gene editing is genetic modification 2.0. Gene editing is directly modifies the genes of the organism itself, so most of them do not exhibit foreign genes. However, the most common gene editing technique, CRISPR-Cas9, introduces foreign genes as the editing tool, and then removes the transplanted foreign genes.

While gene-edited tomatoes were on the market, Japan also approved two types of fish genetically edited with CRISPRtiger pufferfish and red seabream. These fish are genetically edited to accelerate muscle growth. Among them, the gene-edited tiger pufferfish weighs nearly twice that of the ordinary species.

Back in 2019, the United States had used another earlier gene-editing technique to create soybean oil with zero trans fat and introduced it into the market.

Gene-edited foods which have also been approved for sale worldwide by now include soybeans, corn, mushrooms, canola, and rice.

The number of genetically edited foods on the market is likely to increase. Patent applications relating to CRISPR-edited commercial agricultural products have skyrocketed since the 2014/2015 period.

Proponents ofgenetic modification believe this is a method to perfect agricultural produce and solve problems such as pests, droughts, and nutritional deficiencies. But the technology is still a double-edged sword.

Genetic engineering indeed has its benefits in the short term, but it may bring long-term pitfalls, said Joe Wang, molecular biologist. Wang is currently a columnist with The Epoch Times.

Hornless cattle were once the celebrity of the animal kingdom, appearing in news stories one after another.

Many breeds of dairy cattle have horns, but they are dehorned to prevent them from harming humans and other animals, and to save more feeding trough space. To solve the problem of horns, the gene editing company Recombinetics successfully produced hornless cattle with gene-editing techniques many years ago.

The company simply added a few letters of DNA to the genome of ordinary cattle and their offspring didnt grow horns, either.

However, a few years later, an accident happened.

The FDA found that a modified genetic sequence of a bull contained a stretch of bacterial DNA including a gene conferring antibiotic resistance, which has been one of the global health crises in recent years. Scientists arent clear whether this gene in gene-edited cattle will pose a greater risk than expected or not, and the FDA has stressed that its hazard-free. However, John Heritage, a retired microbiologist from Leeds University, told MIT Technology Review that the antibiotic resistance gene could be absorbed by gut bacteria in cattle and could create unpredictable opportunities for its spread.

In fact, this is one of the currently perceived risks of genetically edited foods.

The problem with unexpected accidents in the genetic modification process occurs in GM foods because transgenic techniques cannot control where the foreign gene is embedded in the chromosome.

Kuo used the example of a study that compared the protein of transgenic soybeans and non-transgenic soybeans. These transgenic soybeans were initially embedded with one foreign gene, and should have had only one protein that didnt exist before. However, the comparison showed that there was a difference of about 40 proteins between the two: Half of the proteins were originally present, but disappeared after transgenic modification; the other half were not present but were added after the transgenic modification.

In contrast, emerging gene editing techniques allow for more precise modification of specific genes (pdf). Its like a tailor modifying a section of a zipper by cutting off a specific segment and replacing it with a new one. However, there may be mistakes and unexpected changes in the process of cutting and repairing, and another similar section of the zipper may also be cut off.

Kuo says that this process may have unforeseen side effects; for example, if during this, new allergy-causing proteins or new toxins are produced.

The genetic engineering procedure, and this includes gene editing, has the potential to damage DNA, said molecular geneticist Dr. Michael Antoniou, head of the Gene Expression and Therapy Group at Kings College London, in an interview in April 2022. If you alter gene function, you automatically alter the biochemistry of the plant included within that altered biochemistry can be the production of novel toxins and allergens that is my main concern.

Another major concern with GM foods is herbicide residue.

Most crops, whether genetically edited or genetically modified, have herbicide-resistant genes incorporated into them. This is done so that when herbicides are applied to crops for weed control, the crops themselves wont be harmed.

When planting herbicide-resistant crops, farmers can use herbicides rather liberally. But, long term, the weeds the farmers are targeting become increasingly herbicide-resistant as well, resulting in a cycle of increased herbicide use and resistance.

Since the introduction of herbicide-resistant GM crops in 1996, herbicides have experienced a significant growth in application every year. The herbicides residue in the crops grown are increasing as well.

One of the most widely used herbicides is glyphosate under the trade name Roundup. The International Agency for Research on Cancer (IARC) classifies glyphosate as a Group 2A carcinogen that is probably carcinogenic to humans.

Massachusetts Institute of Technology (MIT) researcher Stephanie Sene and scientific consultant Anthony Samsel said in their study that 80 percent of GM crops, especially corn, soybeans, canola, cotton, sugar beets, and alfalfa, are specifically introduced with glyphosate resistance genes.

In addition to carcinogenic concerns, glyphosate may have more harmful effects. They have collected and reviewed 286 studies and indicated that glyphosate inhibits the activity of an enzyme in the mitochondria of liver cellscytochrome P450which has the ability to detoxify and decompose foreign toxic substances. Moreover, glyphosate also has adverse effects on the gut microbiota.

These effects are not immediately apparent, but in the long run may contribute to inflammatory bowel disease, obesity, depression, attention deficit hyperactivity disorder (ADHD), autism, Alzheimers, Parkinsons, amyotrophic lateral sclerosis (ALS), multiple sclerosis, cancer, infertility, and developmental abnormalities.

An animal study published in Environmental Health shows that long-term exposure to ultra-low doses of glyphosate still causes liver and kidney diseases in rats.

The debate over whether GM food is safe or not has not yet settled. Many advocates of transgenic modification and gene editing believe that people have been eating GM crops for 20-plus years and still there is no evidence that they have caused problems to human health. Other argue they contribute to long term harm that is still being measured.

Kuo said that GM food is not a highly toxic drug causing immediate problems. Health problems can be the result of something cumulative, and hard to relate back to a single food cause. Whether GM foods are the culprit of such health problems has not been proven, nor ruled out.

At present, various countries have adopted an early warning principle for GM foods, stipulating that merchants label their products. It is the consumers decision to purchase them or not.

Will gene-edited food require specific labeling? Some argue that because these foods do not exhibit foreign genes, there should not be such regulation. Kuo believes this is a misleading argument, given that the tool used to edit the original genes were in fact foreign genes, and the method carries the risk that these foreign genes may not be completely removed.

Currently, the regulations for gene-edited foods in various countries are much looser than those for GM foods.

The USDA has consistently stated that gene-edited agricultural products are not regulated. Plant technologists are usually given the green light within months after submitting inquiries to the agency, allowing them to grow gene-edited foods without oversight.

In addition to the United States, Brazil and Australia and other countries have also adopted similar regulatory approaches. European regulations are still more stringent.

Antoniou argues that since these GM agricultural products are not monitored, the unexpected genes that they carry are released into the environment and will cause harm to it. They may also cause harm to the public due to the scientific communitys insufficient understanding of their risks.

Wang said that scientists who support gene editing believe that what they are doing now will also happen in nature, albeit at a slower pace. They simply speed it up. However, humans are not gods and cannot control everything. When humans do such things, the odds of mistakes and danger are definitely higher than what happens naturally, Wang said.

We humans have violated the laws of nature for a long time, Kuo said.

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Camille Su is a health reporter covering disease, nutrition, and investigative topics. Have a tip? kuanmi.su@epochtimes.com

Read more here:
More Foods Will Be Gene-Edited Than You Think - The Epoch Times

USAs Alcor Life Extension Foundation has claimed that it will soon develop the scientific way to rebirth. The company, in USAs Arizona, has preserved the dead bodies of around 199 people only with the hope that once the technique is developed, they will get respite from their illnesses, and they will come back to life. With this hope of these people getting their lives back, Alcor has reserved their dead bodies in nitrogen tanks and the company calls them patients who lost their lives because of illnesses like cancer, paralysis, amyotrophic lateral sclerosis, etc.

Dead bodies preserved this way are called cryopreserved. Among these bodies, the youngest is that of a Thai child, who had lost her life at the age of 2 because of brain cancer in the year 2015. Alcors CEO, Max Moor has said that both of the childs parents are doctors and despite getting multiple brain surgeries performed on her, nothing helped. Hal Finney, who pioneered bitcoin, is also a patient at Alcor. After he lost his life in 2014 due to paralysis, his body is being preserved here.

The process of cryopreservation is highly different, after the legal announcement of a persons death, the blood and other fluids from the body are ejected out, which are then replaced with special chemicals, which prevent the boy from getting damaged, after which the dead body is preserved in extremely cold temperatures like glass.

View original post here:
Alcor Life Extension Foundation preserves 199 dead bodies in nitrogen - Goa Chronicle

Oct. 15Kauai County will give out thousands of free home test kits next week at various neighborhood centers.

The county, in partnership with the state Health Department, is offering about 6, 000 COVID-19 tests kits on a first-come, first-served basis, starting Wednesday. There will be a limit of five tests per person or household.

"Mahalo to our partners with the Kauai District Health Office, the Department of Parks and Recreation, and the Kauai Emergency Management Agency for making these free tests available to our community, " said Mayor Derek Kawakami in a news release. "Home test kits are one of the many tools at our disposal to reduce the risk of spreading COVID."

These COVID-19 tests have an expiration date of November 2022, officials said, but have received a seven-month shelf-life extension from the U.S. Food and Drug Administration. They are set to expire in June.

The distribution schedule will be as follows :

Wednesday9 a.m. to noon, Hanalei Neighborhood Center1 to 4 p.m., Kilauea Neighborhood Center Thursday9 a.m. to noon, Waimea Neighborhood Center1 to 4 p.m., Hanapepe Neighborhood Center Friday9 a.m. to noon, Bryan J. Baptiste Sports Complex1 to 4 p.m., Lihue Neighborhood Center COVID-19 testing is also available islandwide on Kauai, with sites listed at.

Read this article:
Kauai County to hand out thousands of free COVID home test kits - Yahoo News

In this article:

Everyone knows vitamin D -- it's the nutrient we get from the sun. Vitamin D is important for several bodily functions, the main ones being bone and muscle strength and immune function. Still, about 35% of Americans are vitamin D deficient. Research has found that vitamin D deficiencies and diseases like depression, diabetes and cancer are correlated.

Vitamin D is essential. But with winter coming and food source limitations, getting vitamin D isn't always as easy as walking out in the sun. Supplements are used to combat such deficiencies.

A note on units of measurement for vitamin D supplements -- different brands use different units in their marketing. Some measure in micrograms (mcg), while others use international units (IU). The nutritional label typically has both units and the percentage of the daily recommended value. It can get confusing.

Quick conversions that will help you read this list:

Vitamin D supplements span quality, dosage and price point. There are many options, but we narrowed it down to the top eight best vitamin D supplements.

Hum Nutrition Here Comes the Sun is the best overall vitamin D supplement because of its vitamin content and rigorous testing. According to the Hum Nutrition website, this vitamin D supplement boosts mood, maintains your immune system and aids calcium absorption.

It's a non-GMO and gluten-free vitamin D supplement free of artificial flavors, colors or sweeteners. With the serving size of one softgel, you get 250% of your recommended daily value of vitamin D. The vitamin D source was derived from lichen, which makes this vitamin D supplement great for vegans and vegetarians.

Hum Nutrition's vitamin D supplements are sustainably sourced and feature third-party testing.

Price: $19 for a 30-day supply

Vitamin D per serving: 50 mcg or 250% of the daily value

Serving size: One softgel, daily

Hum Nutrition's best features:

Things to consider:

Nature Made vitamin supplements are often my choice for budget shoppers because of the price, vitamin content and company reputation. The Nature Made Vitamin D3 Softgels are available in multiple dosages, so you can choose which works best for you. They have dosages from 25 mcg to a maximum of 250 mcg.

You really can't beat the price of Nature Made. You can get up to a 250-day supply for under $20. Not to mention that the softgels are small and easy to swallow -- and you only have to take one per day.

Price: $16.93 for a 250-day supply

Vitamin D per serving: 50 mcg or 250% of the daily value

Serving size: one softgel, daily

Nature Made's best features:

Things to consider:

If you have trouble swallowing pills, liquid vitamin D options are available the best of which is Pure Encapsulations Vitamin D3 liquid.

It's a small bottle that's easy to transport, which allows you to add it to food anywhere. It's also flavorless. Liquid supplements give you the flexibility that capsule or pill supplements can't. You can also alter the dosage based on seasons and needs. Remember to always pay close attention to how much you're ingesting.

This vitamin D supplement is sourced from lichen. It includes no artificial flavors and is gluten-free and non-GMO. Pure Encapsulation products are third-party tested by organizations like Advanced Laboratories, Eurofins and Intertek.

Price: $29.55 for 22.5 ml bottle

Vitamin D per serving: 25 mcg or 125% of vitamin D of the daily value

Serving size: One drop, daily

Pure Encapsulations' best features:

Things to consider:

For people who need a higher dose of vitamin D, -- those with malabsorption syndromes, osteoporosis or liver failure -- Life Extension Vitamin D3 supplement is a good choice. Most vitamin D supplements have 25 mcg to 50 mcg. The vitamin D supplement from Life Extension offers 125 mcg or 5,000 IU, which is significantly more than other brands.

Given this vitamin D supplement has such a high dosage, it's important to speak to your doctor before taking it. It shouldn't be taken by the average person who doesn't need a huge boost of vitamin D. Life Extension's bottle label advises you to meet with your doctor for regular blood tests to determine your vitamin D levels.

Price: $15.17 for a 120-day supply

Vitamin D per serving: 125 mcg or 625% of the daily value

Serving size: one softgel, daily

Life Extension's best features:

Things to consider:

Many vitamin D supplements happen to be vegan. However, Truvani is the best vegan vitamin D supplement. It's a plant-based and USDA-certified organic supplement sourced from organic lichens that doesn't include additives or processed ingredients. This vitamin D supplement has an average amount of vitamin D at 50 mcg. This vitamin D supplement comes in a small, uncoated tablet. It's easy to swallow, but you can also add it to smoothies or drinks and let it dissolve.

Price: $14.99 for a 30-day supply

Vitamin D per serving: 50 mcg or 250% of the daily value

Serving size: one tablet, daily

Truvani's best features:

Things to consider:

Nordic Naturals Vitamin D3 Gummies are an all-natural supplement free of artificial sugars or additives. With this vitamin D supplement, you get a solid dose of vitamin D without all the extras that gummy vitamins tend to have.

This gummy vitamin D supplement features a wild berry flavor with organic sucrose and fumaric acid sour berry coating. An important thing to note is that while the sugars included are not artificial (organic sugar cane and organic tapioca syrup), there are still two grams of added sugar in this product. It's not the highest I've seen, but it's something to consider if you watch your sugar intake closely. Added sugars are extremely common in the gummy industry -- that's how they get their delicious flavors. Unlike other brands, Nordic Naturals also offers a zero-sugar version sweetened with xylitol.

Price: $12.07 for a 30-day supply

Vitamin D per serving: 25 mcg or 125% of the daily value.

Serving size: 1 gummy, daily

Nordic Naturals' best features:

Things to consider:

Ritual is a well-known vitamin subscription service. It's the best multivitamin with vitamin D because of its traceable ingredients and dense nutrient content. According to Ritual, the Essential Multivitamin helps bone health, brain health and immune function.

Ritual multivitamins include a vegan D3 ingredient made from UK-sourced lichen. With each serving, you get 50 mcg of vitamin D, double what some supplements offer. Since it's a multivitamin, you get additional nutrients like folate and iron.

Ritual supplements are backed by a clinical study that found that the Ritual Essential Multivitamin for Women resulted in a 43% increase in vitamin D levels. It's worth noting that Ritual was involved in the study.

Price: $30 for a 30-day supply

Vitamin D per serving: 50 mcg or 250% of the daily value.

Serving size: two capsules, daily

Ritual's best features:

Things to consider:

Vitamin D is essential for pregnancy as it aids in the development of a baby's bones. Vitamin D deficiencies during pregnancy have been linked to newborns' disordered skeletal homeostasis or fractures. Experts recommend that pregnant women intake up to 4,000 IU (100 mcg) of vitamin D3 daily to prevent preterm births and infections.

Most vitamin D supplements don't offer that much vitamin D3 per serving. However, the FullWell Prenatal Multivitamin does. Developed by a fertility nutrition expert, FullWell supplements are non-GMO and free of common allergens like nuts, dairy, gluten and shellfish.

FullWell Prenatal Multivitamin includes the recommended 4,000 IU of vitamin D and all other nutrients a pregnant person needs -- like vitamins A, V, E and B6. This supplement also offers a hefty dose of pantothenic acid at 2,143% daily value and biotin at 1,429%.

Price: $49.95 for a 30-day supply

Vitamin D per serving: 100 mcg or 667% of the daily value

Serving size: eight capsules, daily

FullWell's best features:

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When choosing the best vitamin D supplements for this list, we considered factors like price, dosage and vitamin D source. We also paid close attention to third-party certifications that the products carry. Due to the limited FDA regulations in the vitamin and supplement industry, third-party testing and certifications are essential to determine product quality and purity. We did not test these products in-house. We relied on customer reviews for things like taste and ease of swallowing.

Our bodies need vitamin D, and as a result, there are a ton of supplements out there with vitamin D in them. From pure vitamin D supplements to multivitamins or prenatal supplements, there tends to be vitamin D in all of them. So which vitamin D supplement should you buy?

When shopping for vitamin D supplements, keep these key factors in mind:

What's the difference between vitamin D and D3?

Vitamin D is broken down into two main forms -- vitamin D2 and D3. The difference is where they come from. Vitamin D2 (ergocalciferol) is found in plant sources like mushrooms or fungi. Vitamin D3 (cholecalciferol is derived from animal sources.

How often should you take vitamin D supplements?

Unlike other nutrients, our bodies store vitamin D in fat cells, which allow them to release it when needed. Many supplements with higher doses of vitamin D are not designed to be taken each day since there likely already is some stored in the body. Taking too much vitamin D can result in stomach discomfort, weight loss and kidney issues. A lower vitamin D supplement is considered safe, particularly if you're deficient in vitamin D.

The information contained in this article is for educational and informational purposes only and is not intended as health or medical advice. Always consult a physician or other qualified health provider regarding any questions you may have about a medical condition or health objectives.

Excerpt from:
8 Best Vitamin D Supplements to Take This Winter - CNET

Cryobiology illustration generated using Midjourney generative AI

Have you ever thought about what would happen if you suddenly need organ transplantation, but no one you know who is willing to donate is a match? An integral part of organ transplantation is, of course, donors and recipients, or people who donate the organs for matching people in need. They are registered within the Organ Procurement and Transplantation Network, an organization that arranges everyone on donor-recipient lists taking into consideration the severity of their illnesses. Their database contains all detailed information on blood and tissue types, organ sizes, medical urgency, and the geographical distance between the donor and the recipient. As soon as there is a newly available organ, a match is found throughout their database and shipped as soon as possible. Or at least thats how the system aims to work.

But there is a hidden player - cold. From Ancient Greece and Rome to modern days, our society has utilized cold in many ways, mostly to preserve food. However, in modern medicine, cold was also found in quite a few applications, such as freezing human sperm and embryos in the process of in vitro fertilization. Intuitively, modern medicine also futuristically looks at cold as a useful agent that could save our lives many years ahead, in the sense of preserving (freezing) our bodies now, and reviving them once we find the cures for untreatable diseases that may have impacted us.

But, coming back to organ transplantation, cold plays a huge role in this process. Once the organ has been removed from the donor's body, it needs to come to the recipient in the exact same functional state. Several external and environmental conditions can severely damage the organ until it's no longer of use. One of the key factors is temperature, which needs to be low enough to slow down biochemical reactions happening in the organ after extraction to prevent further damage. To successfully transport and deliver organs, they need to be kept on ice (a term called hypothermic storage), with an average temperature of +4C. Unfortunately, the heart and lungs can survive on ice for only about 4-5 hours, after which theyre no longer usable. Human organ transplantation requires intense immunological screening of both the donor and the recipient, and this period is usually insufficient to perform it. Finally, 4-5 hours is not enough for an organ to travel from Europe to the United States, for example. It's not even enough to travel within the United States, depending on the ending location, and in many cases, when paired with other logistical constraints, not even sufficient to travel from hospital to hospital. Therefore, geographical location plays a huge role in organ transplantation, and organs that cannot be delivered in a timely manner in optimal conditions will simply be lost. And that's exactly what happens because about 28 thousand organs are wasted in the United States only per year, due to poor performance of currently available preservation methods.

French Blood Bank In Bordeaux. Blood Transfusion Center, Storage Room For Stem Cells In Nitrogen ... [+] 196C. Open Vat Containing Bags Of Stem Cells. Stock Room For Cellular Therapy. (Photo By BSIP/UIG Via Getty Images)

The field of science that investigates the application of cold on biological samples is called cryobiology, whereas the process of using cold to preserve those samples is called cryopreservation. There are quite a few scientific groups, working both in academia and industry, that keep expanding the knowledge in these fields every day. The process of cryopreservation entails many steps, mainly cooling, storage, and rewarming. Each one of these steps can be divided into multiple reactions, and all of them could be performed in multiple ways. It is, however, vital that all of them are performed in an optimal way such that the biological sample that's being preserved does not get damaged, or lose its functionality upon reviving. The main problem in cryopreservation is the formation of ice crystals, that can happen at any step of the way, but mostly when samples are being either cooled to or warmed from subzero temperatures. This is a major issue because the largest part of all biological samples is water. Therefore, many research groups in cryobiology are working on ways to avoid ice crystal formation.

If successful cryopreservation and reviving of complex biological samples, e.g. human organs, was made possible without the interference of ice crystals, organs could be easily transported throughout the world without considering the time it would take to get them to their final destination or be stored for a long time until somebody would need them, as opposed to discarding and losing hundreds of them on a daily basis. Similarly, even if their functionality could be prolonged to a few days instead of a few hours, tens of thousands of human lives could be saved every year. Some researchers dedicated their whole careers to making this happen, and today I will introduce you to one of them.

In my last article on cryopreservation, I had the pleasure of interviewing the group of Dayong Gao, that works on methods to improve reviving of frozen biological samples using single-mode electromagnetic resonance rewarming. Today, I'm interviewing Matthew J. Powell-Palm, an Assistant Professor of Mechanical Engineering and Materials Science at Texas A&M University, and a co-founder of BioChoric Inc. Following in the footsteps of his mentor Boris Rubinsky, he works on understanding the underpinnings of cryopreservation and manipulating the first major part of this process, i.e., freezing itself. The method they are establishing is called isochoric cryopreservation, a technique that could improve transplantation medicine immensely.

Cryobilogy in cancer

The History of Cryopreservation: Major Breakthroughs

By providing you a little bit of historical context, well have a look over the major breakthroughs that happened in the field of cryobiology, and that instigated the modern use of cold in medicine. The start of the modern field of cryobiology is thought to have happened in 1948, when Christopher Polge discovered the cryoprotective effects of glycerol, a cryoprotective agent (CPA) that prevents ice crystal formation through the creation of bonds with free water molecules. Since then, a huge aspect of cryobiology and cryopreservation technologies was that we can modulate a given system's chemistry by involving CPAs, which could, in theory, allow us to preserve a live biologic sample for a long time. Many more CPAs, like dimethyl sulfoxide (DMSO), appeared on the scene afterwards, revolutionizing the subfield of human sperm cryopreservation. In 1972, scientists Peter Mazur, Stanley Leibo, and David Whittingham published evidence of the first-ever successful cryopreservation of mammalian embryos using slow-freezing. Eleven years later, the first-ever human embryo was cryopreserved.

A turning point in cryobiology happened in the 1980s, the so-called golden era of cryopreservation. Building on seminal early work by Father Basile J. Luyet, a Catholic priest and professor who helped to establish the thermodynamic foundation of modern cryobiology, Gregory M. Fahy and William R. Fall introduced the process of vitrification to medical cryopreservation. Vitrification is a process of rapid cooling of liquid medium until it becomes a glass-like non-crystalline amorphous solid. It requires the protective effect of CPAs, which lower the freezing point of water, as a major part of biological systems. In its vitrified state, water is locked in place, preventing the formation of ice crystals, and the entire sample becomes a glass-like solid. Vitrification is used widely today in the cryopreservation of very small biological samples (specifically in in vitro fertilization and other reproductive applications), and many cryobiologists believe it could eventually be applied to freeze any biological materials, even organs and whole organisms.

Human kidney frozen in ice cube, 3D rendering isolated on white background

Using vitrification, many research groups have already been able to successfully preserve and revive different cells and tissues, showing that there is major potential in cryopreserving and reviving organs as well. One of the major focus in cryobiology research is, in fact, centered around the process of vitrification and how much and which CPAs to add during this stage, or how to remove them in the rewarming stages. But, so far, CPA-aided vitrification only enabled the routine preservation of cells and cell suspensions and failed to produce any clinically translatable technique on how to preserve any complex biological systems like organs outside of the human body.

Isochoric Cryopreservation: Out With the Old, In With the New?

Methods in cryopreservation havent changed much in the last few years but there is a different approach currently available called isochoric cryopreservation. The term stands for cryopreservation of biological tissues at a constant volume, versus the more traditional way of cryopreservation that's done at constant pressure, called isobaric cryopreservation. During isochoric preservation, the cooling process happens in a confined, constant-volume chamber, representing one of the biggest differences between isochoric and isobaric conditions. Another difference is minimized role of CPAs, which are very much needed in the classical isobaric cryopreservation, but not in several modes of isochoric cryopreservation. The advantage of isochoric freezing is that it completely avoids the question of the toxicity associated with CPA usage as well as the amount of CPAs needed to be present in the biological sample you might want to freeze. Even if there is a need to use CPAs, their concentrations would be dramatically decreased. Under isochoric conditions, a biological sample is confined within a container with high rigidity and strength, usually made out of titanium. The container is completely absent of the bulk gas phase, and is denied any access to the atmosphere, which changes both the thermodynamic equilibrium and the ice nucleation kinetics within the system inside.

Isochoric cryopreservation is a technique conceived initially by Boris Rubinsky, a Professor at the University of California at Berkeley. Prof. Rubinsky obtained his Ph.D. at MIT in 1981 and has been engaged in the field of cryobiology ever since. His major research interests include heat and mass transfer in biomedical engineering and biotechnology and, in particular, low-temperature biology, as well as the development of bio-electronics and biomedical devices for clinical purposes. He has also pioneered in the fields of medical imaging, cryoablation, and non-thermal electroporation. Prof. Rubinsky has been involved with more than 470 peer-reviewed scientific papers since the beginning of his career and holds more than 30 US-issued patents.

The aim of isochoric cryopreservation at Prof. Rubinsky's group is not strictly preservation of biological samples (to be revived) per se, but rather about further developing the technique to offer the world a chance for a more successful general process of cryopreserving biological samples and decreasing the using toxic CPAs. Some of their latest research includes the creation of a quantitative approach to develop a general framework for the design of metastable supercooling protocols which incorporate the phase transformation and biochemical kinetics of the system. You can find the paper here. The group has also played with carbohydrate polymer protectants, as opposed to the small-molecular weight chemical ones mostly in use nowadays, and found that they can be used to manipulate the metastable-equilibrium phase change kinetics of the system at subzero temperatures. This approach has revealed that a carbohydrate polymer can be used to help modulate the stochasticity of ice nucleation in the supercooling system, which is important to designing supercooled biopreservation protocols for practical use. This research can be read here.

It seems the group is really striving to develop and optimize an application of supercooling and freezing techniques that could be used in biomedical devices already today. Some of Prof. Boris Rubinsky's technologies were already used to treat tens of thousands of patients, and the companies he founded were acquired by the big fish, such as Cryomedical Sciences which became a $300 million NASDAQ company. A new name in the field of isochoric cryopreservation is eager to follow in these steps, and to further develop the field in his own way: Matthew J. Powell-Palm.

Future Players in Cryo-thermodynamics: Professor Matthew J. Powell-Palm

Matt Powell-Palm is one of Boris Rubinsky's former PhD students and a leader in the field of isochoric cryopreservation. He is currently an Assistant Professor of Mechanical Engineering and Materials Science at Texas A&M University, and a co-founder of BioChoric Inc. (along with his former PhD supervisor), a medtech startup that is working on transforming transplant medicine by developing methods to prolong organ preservation. He obtained his Master's degree in 2016 at Carnegie Mellon University under the supervision of Jon Malen, and his Ph.D. in 2020 at UC Berkeley.

Currently, a central focus of Matt's research is within the field of isochoric thermodynamics and cryopreservation. His expertise revolves around the applications of isochoric supercooling and vitrification protocols and devices to improve organ preservation, conserve endangered marine animals, and improve global food storage and transportation. Even though he completed his Ph.D. only two years ago, he's already established himself as one of the leaders in the field of isochoric thermodynamics and cryopreservation with more than 25 published peer-reviewed scientific papers and numerous patents. I was honored to share the online space for some time with Matt and pick his brain on all things cryo, plus ask some additional futuristic questions.

Alex Zhavoronkov, PhD interviewing professor Matthew Powell-Palm via Zoom, September, 2022

First, I wanted to see what Matt's perspective was on different terms in cryobiology, and what he considers the differences between them.

Alex: Can you describe the differences between cryonics, cryobiology, and cryopreservation?

Matt: Cryopreservation is the application of cryobiology, and the biggest difference between it and cryonics is the end goal. The field of cryopreservation is not particularly interested in existential or societal aspects of life prolongation and is solving daily problems in medicine, conservation biology, agriculture, and in any application where the elongated shelf life is important. Cryonics is the application of cryobiology where the end goal is to prolongate a human life by freezing and reviving it in the future.

Alex: Can you talk about your current research and, specifically, the concept of isochoric cryopreservation?

Matt: Looking back on the many successes and failures of modern cryopreservation, I have been asking myself the past few years if there are any new non-chemical ways in which we can manipulate the thermodynamic behaviors of water to achieve the goal of preventing ice crystal formation below the systems melting point, which is the main problem in cryopreservation.

The umbrella technique the Rubinsky Lab has come up with leverages the effect of confinement or constant volume thermodynamic properties to manipulate phase transitions and equilibria of water. In the world around us, we are always in communication with the atmosphere as this constant and infinite pressure reservoir, and the core premise of isochoric cryopreservation processes is that we may be able to affect the phase equilibria and kinetics of water and ice by denying them access to this constant atmospheric pressure. When we do that, the natural variables that describe their existence are now constant volume and temperature, not pressure and temperature. When we confine the volume of a given system, it has a huge effect on the relationship between water and ice. We all know water expands almost 10% upon freezing, and weve all left a bottle of water or beer in the freezer only to come back and find it exploded. So lets imagine what would happen if instead of having liquid in a glass bottle, we held it in an unbreakable titanium flask. Ice will form and try to expand, but now it can't break the container or push the water out. What happens? Ice will start to expand, but the flask won't break and will instead push back on the contents within, pressurizing the growing ice and the remaining water. As a result, only a small portion of the liquid will end up as ice, even at temperatures well below the freezing point.

And isochoric conditions affect not only the equilibrium between water and ice, but also the metastability of water, the vitrification process of water, and the ice nucleation and growth process. So we are working on a broad suite of thermodynamic techniques that arent dependent on chemical intervention but enable us to reach sub-zero temperatures without ice formation in a stored biologic, which opens up many new avenues for exploration in cryobiology.

Alex: Among the classical isobaric approaches used in cryopreservation with antifreeze agents, vitrification, and rapid reheating, how is isochoric preservation better?

Matt:

You can think of the isochoric effect as being a value-add to any system. Speaking generically, our data and research suggest that if you take any classical technique or system and conduct the same protocol not under atmospheric pressure, but instead under isochoric conditions, you will encounter a lower chance for ice crystal formation. For conventional vitrification for example, you need incredibly high concentrations of cryoprotectants, usually 7 to 10 mol/L, or up to 40-50 % of the weight ratio. By using isochoric conditions, we can relieve some of the work that the chemistry needs to do in aiding glass formation, facilitating the same process of vitrification using a lower concentration of cryoprotectants, but under isochoric conditions. Similarly we can supercool metastable systems with higher reliability by confining them, we can hold equilibrium systems in a passively pressurized ice-free state, and so on.

Ill note too that a lot of the classical cryobiology literature and techniques have focused on ultra-low temperature preservation that targets months or years-long preservation, but there are all kinds of pressing medical cryobiology problems that dont necessarily require that, the most obvious being full organ preservation, where shelf-life extension on the order of even a single day would be transformative. So theres been a notable shift in the last decade towards what the community calls high subzero methods, which operate in the 0 20C range and leverage processes that aim to be much less physically and chemically intensive on the biologic than something like vitrification. Were finding that isochoric techniques can be particularly useful in this domain too, because you enter the realm where totally-CPA free isochoric supercooling or isochoric freezing protocols are very possible.

Alex: What about rapid reheating by using microwaves? How does the isochoric approach help with this?

Matt: Our goal is to build our protocol so that we ultimately wont need rapid reheating, which is required to escape the high probability of ice crystal formation when rewarming biological samples. If we can decrease the probability of ice crystal formation across the board, we would decrease the need to use rapid reheating. For example, and although I can't talk about it in too much detail, we are collaborating with the Smithsonian Conservation Biology Institute on vitrifying whole fragments of endangered corals under isochoric conditions, which has never before been achievable. In preliminary data, we are able to reheat the system without problems at a ballpark rate of 100s of degrees C per minute. The more sophisticated electromagnetic heating techniques achieves warming up rates of thousands of degrees and up in small systems, and those methods are indeed very cool, but so far unneeded for our systems. Ill note too that another aspect of the rewarming challenge is heating the system without building up significant thermal stress, which can lead to cracking throughout the sample because of uneven heating. One advantage that the isochoric system appears so far to offer is that physically confining the volume can help stabilize the system against cracking. If your system is open to the atmosphere, as it warms, the outermost layer that's open to the environment can expand freely, and cracking can happen easily. In the isochoric system, the boundaries of the sample are constrained, and it can help with reducing thermal cracking.

Matt's answers really intrigued me. I have been looking at cryopreservation through the eyes of cryonics and improving medicine by being able to extend the time until we find cures for untreatable diseases, which would imminently save so many human lives. However, it seems one part of the field, which Matt is intensively developing with his colleagues, could help to save so many lives in the present time very soon. It seems like a real, graspable possibility.

However, this made me wonder about the field of cryopreservation I have been interested in for months now. We saw some major breakthroughs in the field a long time ago, but lately, it seems as if the progress has been really slow. Is it because the field has been focused on the complicated process of vitrification by using cryopreserving agents too much, or is there something else at play? I was interested in what Matt had to say about this.

Alex: Clearly, the field of cryopreservation has been around for quite soe time. Why did it not yet pick up?

Matt: This is a fascinating question thats obviously affected by many different factors both historical and contemporary, but one of the biggest as always is funding, plain and simple. In the 90s and early 2000s, there was vanishingly little money available for research on cryopreservation, and what money there was was sort of narrowly focused. In the last decade however, cryopreservation, which we now include under the larger umbrella of biopreservation, has become something of a space race, and funders as varied as NIH, USDA, DOD, and even NASA are now giving out money for low-temperature biopreservation research. For example, NASA is looking for ways to protect astronauts in the theoretical manned missions to Mars. Even though using cryopreservation techniques to achieve goals like that seemed like sci-fi only a few years ago, we are now seeing more and more adventurous cryopreservation ideas getting funded, and funded well, and this has enabled the modern cryobiology field to start operating at the pace expected of a cutting-edge, super-impactful branch of science.

Alex: What happened in the last 5 years in cryopreservation research that may result in a major breakthrough in industrial applications?

Matt: Oh yeah, the last 5 years have been huge. Im lucky to get to see watch this progress unfold from both the academic angle, as a professor, and the industrial and clinical angles, as a startup founder. The suite of core technologies driving cryopreservation these days has just exploded in the last half-decade or so, driven by key advances in our understanding of aqueous metastability and supercooling of bulk volume liquids, uses of electromagnetic effects and nanoparticles for rapid and uniform warming, new thermodynamic configurations like isochoric, and many more. These fresh approaches are driving work in all sorts of new applications, and bringing new interdisciplinary physical science angles to the field.

Supercooling alone is a potentially transformative technology for large clinical applications, e.g. to extend the shelf-lives of transplantable livers, hearts, kidneys, etc. Id put my money on that technique seeing the light of day in the clinic within the next 5 years, as some kind of self-contained supercooling device. In my company, we have an isochoric supercooling technique that I think can be ready for pre-clinical trials very soon, though I can't say too much there. But the potential public health benefit of stable supercooling is just tremendous. I mean, if you could extend the preservability of a heart by just 4-8 hours, you might save a thousand lives next year. Extend it by a day or two and you could potentially be saving tens of thousands of lives around the globe.

As a field, we don't need technologies that will take ten more years to develop and will enable indefinite storage of a human heartwe need technologies that will take ten more months to develop and will enable storage of a heart for just long enough to get it from the donor to the recipient!

Although Matthew didn't point it out now, he is also doing a lot of work on preserving and extending the shelf life of food, which is another pressing societal issue, given the rising problems of food waste in some regions of the world, and the lack of food in other regions at the same time. In one of the groups latest research papers, isochoric supercooling and freezing have been applied to freshly harvested pomegranate, with its shelf-life being successfully extended for a month. You can read the publication here.

At his young age, Matthew is already wearing two hats (as he candidly points out), one of an academic professor and researcher, and the other as a co-founder and owner of a start-up company called BioChoric Inc. The company carries on with its research on isochoric preservation and aims at putting applicable devices and methods on the medical market as soon as possible, with everything being rooted in peer-reviewed and solid-proof research. Matt shared with me what the first days of starting the company looked like, and what their main future goals are.

Alex: When did you start BioChoric Inc. and what drove you to it?

Matt: We started the company in 2020 during the COVID pandemic. It was a spinout out of UC Berkeley, with me and Boris Rubinsky as founders, and the impetus was a crop of data we got on the effects of isochoric conditions on the supercooling of water, which suggested to us that an isochoric supercooling approach may be immediately applicable to organ and tissue preservation. We have a couple of integral patents and papers that describe the premise that, by confining the system, we can stabilize water in a metastable supercooled state, and predict the behaviors of this state in a rigorous quantitative sense, which has so far proven very difficult in unconfined systems.

The underlying philosophy of BioChoric Inc. is the obligation we feel to make rapid if incremental progress in full organ preservation. The degree of donor organ waste and the number of people dying on organ transplanting lists every day is huge, and that made us look at everything with a more clinical perspective. That's what we're pushing forward with BioChoric, even though the company is very small for now. One unique thing about the company is that it represents most of the thermodynamic expertise surrounding isochoric systems in the world today, and we rely heavily on interdisciplinary academic collaborations to help us further build the confidence and evidence we need to start pushing our techniques to clinical markets. We haven't taken any outside funding and it's fully internal equity, even though we've been approached by investors several times. We want to make sure we are scientifically sterling, peer-reviewed, bullet-proof before we start trying for the clinic.

One of the side hats BioChoric wears is also building isochoric biopreservation platforms and devices for other labs interested in advancing the science, and the small profit we generate from that helps to sustain our early R&D efforts.

It seems Matt is fully focused on improving human lives in the sense of prolonging the time transplantation-ready organs can be preserved, and that's the main goal of BioChoric. However, Matt and Boris's company is not the only one out there that offers cryo-products, although it may be the only one with a focus on isochoric cryopreservation, at least for now. Let's see what Matt thinks about how his company compares to similar ones in the field of cryobiology.

Alex: How do you compare and compete with companies like Lorentz Bio or X-Therma? When do you think BioChoric Inc. will be ready to fundraise and go industrial-scale?

Matt: I think there are many great young companies popping up in this space, but I'm glad you bring up Lorentz Bio because it has sparked quite a bit of chatter in the community, and they're taking an approach opposite to ours I think. My generic observation is that they have tackled raising the big money first, presuming they can fill in the scientific blanks later. In our case, its the scientists who have built the company, and built it on a core piece of new science, and were presuming we can fill in the money blanks later! Both fine ways to approach the problem. But personally, Im not really in the business of speculation or gambling I'm here to make sure were producing rock-solid, air-tight science, and the fundraising aspects don't worry me as much. Maybe that's just my academic side coming out. I think historically though, companies with really high checkbook-to-scientist ratios often end up coming to companies with really high scientist-to-checkbook ratios, like ours, to license our scientifically-established techniques and products. So suffice it to say, were focused on the science first and everything else second, and we're shooting for both fundraising and expansion to industrial scale in the next two years.

As my final question, I asked Matt the same futuristic question I asked Dayong Gao's research group at the University of Washington's Center for Cryo-Biomedical Engineering and Artificial Organs in my first article on cryopreservation, which you can read here. Matt was brave enough to offer me a timeline in which we could see some real breakthroughs in cryonics, as opposed to only preservation.

Alex: When do you think we will be able to see isochoric cryopreservation being used to cryopreserve and revive a small mammal?

Matt: Interesting question! I would say within the next 5 years, we will certainly see isochoric preservation of endangered marine species. Marine biodiversity is such an unbelievably urgent problem, and we are thinking about expanding our research on coral to other marine organisms in the next few months. If things continue to go well, we may be looking at trying to deploy field-ready isochoric devices at every marine research station on Earth, as bombastic as that sounds! The problems there are just too pressing to wait. On the human organ scale, I think we will see the preservation of organs extended to at least single days within the next 5 years. And I also want to take this opportunity to give a shoutout to each and every research group working on this problem right now, because the many often divergent results from differing corners of the field each move us all forward.

I admittedly havent thought much about preserving small live mammals, so I cant speculate in a properly scientific fashion, but Ill speculate for fun! Current approaches would require us to preserve each organ of the mammal separately because the preservation process gets more complicated the more complex you go. Based on the progress in the last 5 years, we will probably see a supercooling approach to preserve every major organ separately within the next 5 years. I don't know what happens at one step higher if you would want to preserve a multiorgan construct, and what would be different about it in comparison to just one organ. The relationship of these animals with air is also more complex than with marine animals that live submerged in a liquid anyway. But as a cop-out, Ill go ahead and say that the timeline will once again depend really acutely on potential increases in funding, and it will depend on which aspects of the field will get the most funding. So, I would speculate we could see a small mammal preserved and revived in about 20 years if the funding goes in that direction. But in my opinion, there is much more pressing research to be done.

With such young and bright-minded scientists led by the field's giants, like the combination of Matthew J. Powell-Palm and Boris Rubinsky, cryopreservation is definitely looking at several major breakthroughs coming from all areas of the field in the next few years. Also, as Matt also smartly pointed out, progress coming from different areas of cryopreservation actually helps developing all areas of cryopreservation, as the complex process of cryopreservation itself is made of various tightly-bound and regulated steps that cannot work alone.

See more here:
Think Outside The (Titanium) Box: Isochoric Cryopreservation Could Save Lives - Forbes

A recent report commissioned by the Chamber of Commerce for Greater Philadelphia and researched by economic consulting firm Econsult Solutions, Inc. ranked the city No. 2 overall for cell and gene therapy (CGT) hubs. The analysis evaluated 14 US CGT hubs across five categories, including research infrastructure, human capital, innovation output, commercial activity, and value proposition. The only region to eclipse Philadelphia was Boston. New York and San Francisco ranked third and fourth, respectively.

According to the report, Greater Philadelphia researchers have been awarded at least $1 billion in NIH funding in each of the past five years. Focusing in more closely on research projects related to CGTs, more than $317 million in NIH funding has been awarded to Philadelphia investigators during that time period. Funding for CGT comprised 6% of total NIH funding in Philadelphia compared to a range of 0.7% to 5.2% in the comparison regions.

The volume of research funding is an indicator of the potential pipeline of discoveries and innovation that can be generated from basic academic research in the coming years.

In addition to the Pennsylvania citys second-place showing in CGT hub prowess, the Chamber of Commerce for Greater Philadelphias CEO Council for Growth, along with its partners in the Cell and Gene Therapy Initiative, have released a new video (https://bit.ly/3EnUfXU) on the Philadelphia regions connected CGT startup ecosystem. Titled Greater Philadelphia: Discovery Starts Here, the 90-second video animation shares a snapshot of several of the regions research institutions and a number of the CGT-focused companies that have licensed technologies.

The video highlights five of the regions leading research institutions: Childrens Hospital of Philadelphia, Temple University, Thomas Jefferson University, University of Pennsylvania, and The Wistar Institute. It then shifts the focus to 15 companies that have direct links to one or more of those five research institutions. The video also distinguishes the organizations in four categories: emerging, privately held, publicly traded, or acquired.

The 15 CGT companies highlighted in the video include: Adaptimmune; Aevi Genomic Medicine, Inc. (acquired by Avalo Therapeutics, Inc.); Cabaletta Bio; Carisma Therapeutics; Cartio Therapeutics; Imvax; INOVIO; Interius BioTherapeutics; KOP Therapeutics; Passage Bio; Renovacor; Scout Bio; Spark Therapeutics, a member of the Roche Group; Verismo Therapeutics; and Virion Therapeutics.

For more information on the video and the Chamber of Commerce for Greater Philadelphias CEO Council for Growth, the full press release can be found here.

Link:
Philadelphia Shines in Cell and Gene Therapy Research Pursuits - Applied Clinical Trials Online

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The success of cell therapy products, including CAR-T cell therapy, is significantly dependent upon the material selection strategies set in the early stages of development. These strategies encompass raw materials (i.e., ancillary) throughout the production process. To facilitate the translation into clinical trials and beyond, developing a raw material selection strategy that considers the end goal is critical. Most strategies emphasize the use of the highest grades of materials available as early as possible; however, this may not always be feasible. Accounting for material-grade transitions can help balance both performance and costs when moving onto the next stage.

Raw and ancillary materials refer to materials that are used during the production process but that are not present in the final cell therapy product. Overall, these materials are not explicitly regulated. However, there are guidelines that provide recommendations on material selection due to its significant impact on safety, purity, and potency in the final product. When selecting raw materials for use, cell therapy manufacturers should deliberate on the biosafety characteristics and the risks each material could introduce.

For early-stage research and development of cell therapy, research-use only (RUO) materials are most commonly used. However, RUO materials do not meet the regulation of clinical phases. The FDA recommends using FDA-approved or clinical-grade materials; therefore, the best option is utilizing good manufacturing practice (GMP) or current GMP (cGMP) grade materials.

GMP products are manufactured under a stringent quality management system that is significant costlier than RUO counterparts. Implementing GMP products in the early preclinical, or even clinical, stages may not always be feasible. Therefore, transitioning between RUO products to GMP products is important. Seeking qualified, reliable, and consistent raw material manufacturers is the material-selection strategy and is a key component in developing a cell therapy product.

ACROBiosystems offers a wide range of raw materials and tools needed for cell therapy products, including cytokines, nucleases, and activation beads. We provide several grades of materials, including both premium and GMP grades. Our premium-grade proteins originate from the same clone, sequence, and expression system as our GMP products. The main difference is the available documentation provided with our GMP products. This means developers utilizing our premium-grade products can easily transition into GMP products for use in CMC or clinical stages without more comparability studies of the manufacturing process.

Cell therapy products are a type of living drugs that are under increasing scrutiny from regulatory bodies due to both their potential and safety concerns. As such, there are numerous pitfalls when developing a cell therapy product. Having a long-term material- and process-selection is critical to translating a product into clinical and commercialization phases. To assist our customers throughout the entire development process, ACROBiosystems has developed a series of high-quality cell therapy products that maximize bioactivity and potency while preserving research budgets.

For additional information: http://www.acrobiosystems.com

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Selection and Evaluation of Ancillary Materials for Cell and Gene Therapy Research - Inside Precision Medicine

BEIJING & BURLINGTON, Mass.--(BUSINESS WIRE)--CANbridge Pharmaceuticals Inc. (HKEX:1228), a leading global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that data from its gene therapy research agreement with the Horae Gene Therapy Center, at the UMass Chan Medical School, was presented at the 29th European Society of Gene and Cell Therapy Annual Congress in Edinburgh, Scotland, today.

In an oral presentation, Guangping Gao, Ph.D., Co-Director, Li Weibo Institute for Rare Diseases Research, Director, the Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems and Penelope Booth Rockwell Professor in Biomedical Research at UMass Chan Medical School, discussed the study that was led by the investigator Jun Xie, Ph.D., and his team from Dr. Gaos lab, and titled Endogenous human SMN1 promoter-driven gene replacement improves the efficacy and safety of AAV9-mediated gene therapy for spinal muscular atrophy (SMA) in mice.

The study showed that a novel second-generation self-complementary AAV9 gene therapy, expressing a codon-optimized human SMN1 gene. under the control of its endogenous promoter, (scAAV9-SMN1p-co-hSMN1), demonstrated superior safety, potency, and efficacy across several endpoints in an SMA mouse model, when compared to the benchmark vector, scAAV9-CMVen/CB-hSMN1, which is similar to the vector used in the gene therapy approved by the US Food and Drug Administration for the treatment of SMA. The benchmark vector expresses a human SMN1 transgene under a cytomegalovirus enhancer/chicken -actin promoter for ubiquitous expression in all cell types, whereas the second-generation vector utilizes the endogenous SMN1 promoter to control gene expression in different tissues. Compared to the benchmark vector, the second-generation vector resulted in a longer lifespan, better restoration of muscle function, and more complete neuromuscular junction innervation, without the liver toxicity seen with the benchmark vector.

This, the first data to be presented from the gene therapy research collaboration between CANbridge and the Gao Lab at the Horae Gene Therapy Center, was also presented at the American Society for Cellular and Gene Therapy (ASGCT) Annual Meeting in May 2022. Dr. Gao is a former ASCGT president.

Oral Presentation: Poster #: 0R57

Category: AAV next generation vectors

Presentation Date and Time: Thursday, October 13, 5:00 PM BST

Authors: Qing Xie, Hong Ma, Xiupeng Chen, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Qin Su, Phillip Tai, Guangping Gao, Jun Xie

Abstracts are available on the ESGCT website: https://www.esgctcongress.com/

About the Horae Gene Therapy Center at UMass Chan Medical School

The faculty of the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited disease for which there is no cure. We utilize state of the art technologies to either genetically modulate mutated genes that produce disease-causing proteins or introduce a healthy copy of a gene if the mutation results in a non-functional protein. The Horae Gene Therapy Center faculty is interdisciplinary, including members from the departments of Pediatrics, Microbiology & Physiological Systems, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology. Physicians and PhDs work together to address the medical needs of rare diseases, such as alpha 1-antitrypsin deficiency, Canavan disease, Tay-Sachs and Sandhoff diseases, retinitis pigmentosa, cystic fibrosis, amyotrophic lateral sclerosis, TNNT1 nemaline myopathy, Rett syndrome, NGLY1 deficiency, Pitt-Hopkins syndrome, maple syrup urine disease, sialidosis, GM3 synthase deficiency, Huntington disease, and others. More common diseases such as cardiac arrhythmia and hypercholesterolemia are also being investigated. The hope is to treat a wide spectrum of diseases by various gene therapeutic approaches. Additionally, the University of Massachusetts Chan Medical School conducts clinical trials on site and some of these trials are conducted by the investigators at The Horae Gene Therapy Center.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 11 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme. CANbridge is also building next-generation gene therapy development capability through a combination of collaboration with world-leading researchers and biotech companies and internal capacity. CANbridges global partners include Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School and LogicBio.

For more on CANbridge Pharmaceuticals Inc., please go to: http://www.canbridgepharma.com.

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CANbridge-UMass Chan Medical School Gene Therapy Research in Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual...

CBER maps modernization plan to handle surge in research and applications.

FDAs Center for Biologics Evaluation and Research (CBER) is updating how it manages a growing volume of cellular and gene therapy development programs, seeking added resources and revisions in its oversight of these cutting-edge therapies. Most visible in the elevation of CBERs Office of Tissues and Advanced Therapies (OTAT) into a new super Office of Therapeutic Products (OTP). The change aims to improve functional alignment, increase review capabilities, and add expertise on new cell and gene therapies by establishing multiple branches and divisions in the expanded regulatory unit, as announced in the Federal Register on Sept. 28, 2002.

Stated goals are to help CBER address the substantial growth in innovative, novel products that present new scientific, medical and regulatory challenges that require changes to its structure, including strategies to advance the Regenerative Medicine Advanced Therapy (RMAT) program. The added resources are needed to oversee more than 2000 development programs involving cellular and gene therapies, many involving innovative testing and manufacturing processes. This soaring workload has over-taxed CBER staffers, resulting in serious difficulties in retaining and hiring capable scientists.

The structural changes at CBER reflect agreed-on plans to hire new staffers with funding from recently reauthorized user fee programs. The PDUFA VII commitment letter calls for an additional 132 new hires for CBER in this coming year and another 48 employees the following year, most to support cell and gene therapy reviews at OTP. The reorganization plan calls for OTP to have seven officesfor therapeutic products, clinical evaluation, review management, pharmacology/toxicology, and two for CMCfor gene therapy and for cellular therapy and human tissues. There will be 14 divisions and 32 branches within those offices, providing attractive supervisory opportunities for both new and experienced staffers.

These changes come in the wake of FDA approval of two new gene therapies that have drawn wide attention for both their therapeutic potential and for million-dollar price tags. Bluebird bios Zynteglo was approved by FDA in August for patients with beta thalassemia, an inherited blood disorder causing serious anemia. That was followed a few weeks later with approval of Bluebirds Skysona to treat a rare neurological disorder afflicting young boys. Zynteglo carries a $2.8 million price tag, Skysonas list price is $4 million, but both therapies are expected to target fewer than 1500 patients, limiting the overall cost impact for the US healthcare system. A greater spending effect would come from FDA approval of a new treatment for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics, which plan to begin a rolling review by FDA in the coming months. The important potential benefits of these treatments, along with concerns about their impact on healthcare spending and access, speaks to the need for a highly capable and sufficiently resourced FDA oversight program.

These developments also highlight the importance of sound testing and production methods for therapies made from living organisms, which are inherently variable and difficult to control and measure to assure product safety, identify, quality, purity, and strength. The surge in applications from a broad range of firms, moreover, has made it difficult for CBER staffers to schedule formal meetings with each sponsor seeking advice on how best to perform manufacturing and testing processes. And publishing new guidance on these changing and emerging issues also takes time and resources.

In response, FDA looks to engage a broad range of sponsors on topics related to product development through a series of virtual town hall meetings. The first was held Sept. 29, 2022 and addressed how manufacturers should describe and inform FDA about chemistry, manufacturing, and controls (CMC) in applications for gene therapies. Wilson Bryan, OTAT (now OTP) director, opened the session by describing plans for establishing OTP as a super office to increase review capabilities and enhance expertise on gene and cellular therapies and set the stage for OTP branch chiefs to field a broad range of queries, ranging from basic CMC policies for various stages of development, to the scope of potency assays and impact of delivery devices on dose potency and quality [a recording of the town hall meeting is available at the FDA events link].

Main topics were comparability testing, assays for product characterization, and process controls. OTP staffers emphasized the importance of determining process requirements early in development to avoid late changes and analytical method variability that could raise uncertainties likely to delay clinical trials. Products with complex mechanisms of action, they advised, stand to benefit from early product characterization and potency assay development. And developers of gene therapies should use multiple production lots during a clinical study to ensure product consistency and quality, even for treatments for very small patient populations.

Manufacturers raised questions about differing CMC issues between early Phase I and late-stage clinical trials and voiced concerns about product characterization related to autologous cell-based gene therapies. A main theme from FDA was the importance of sponsors establishing a well-controlled manufacturing process and qualified analytical testing well before administering any new gene product. While CBER plans to issue guidance on manufacturing changes and comparability for cellular and gene therapy products, the information provided at this session provides unofficial guidance for implementing changes in product manufacturing and the scope of comparability assessments and development studies expected to support such changes.

Jill Wechsler is Washington editor for Pharmaceutical Technology.

Excerpt from:
FDA Expands Oversight of Cell and Gene Therapies - Pharmaceutical Technology Magazine

In this interview, Arnaud Delobel, the R&D and Innovation Director at Quality Assistance talks to AZoM about the emerging field of gene therapy with adeno-associated viruses (AAV).

Our organization, Quality Assistance, is a contract research organization. Our work provides scientific, regulatory, and technological expertise to our clients, who develop innovative human medicinal products. Our work is strictly compliant with US and EU regulations.

Regarding analytical protocol and methods, we offer customized solutions. When it comes to the acquisition and interpretation of data, Quality Assistance provides a wide array of cutting-edge equipment, which can then be included in the regulatory dossier. Our work is performed in a single site based in Belgium.

With 40 years of experience, Quality Assistance is a stable, reliable analytical partner working hard for our customers.

The Emerging Field of Gene Therapy with Adeno-Associated Viruses - Wyatt and Quality AssistancePlay

To support the development of innovative products, Quality Assistance offers our customers the benefit of our analytical expertise to determine drug safety, quality, and efficacy. This is where Quality Assistance's customized solutions are essential, including the development and validation of specific methods for each product and the design of analytical protocols.

Quality Assistance also offers characterization services, stability studies, battery testing, PK biomarker, and immunogenicity studies.

With our strong product-dedicated expertise, Quality Assistance has worked for 40 years on producing and managing new chemical entities, along with working on peptides and oligonucleotides. Over the last two decades, the company has worked on recombinant proteins such as monoclonal antibodies and antibody-drug conjugates.

Our services for emerging markets, such as mRNA-based therapies, cell and gene therapies, and viral vectors, have been a focus for development over the last few years.

There is definitely an increasing demand for analytical methods for characterizing AAV therapies. Over the last few years, most projects we handled that were in the research phase switched to the clinical phase. With this switch, robust analytical methods, offering results that can be included in regulatory dossiers, became essential.

To keep up with this demand our organization invested inWyatt SEC-MALS system, which is a powerful tool for characterizing AAVs. We can now provide our customers with reliable, validated methods, and results that can be included in regulatory files.

Image Credit: ShutterShock/Kateryna Kon

We use Wyatts DAWN multi-angle light scattering detector with size-exclusion chromatography (SEC-MALS). We also use Wyatts Eclipse asymmetric-flow field-flow fractionation (AF4) instrument along with a multi-detector system for the separation and characterization of AAVs (MD-AF4). Our multi-detector system includes a Wyatt Optilab refractive index (RI) detector, a UV detector, and a DAWN multi-angle light scattering (MALS) equipped with online dynamic light scattering (DLS) detection module for in-depth analysis.

Our organization has over a decade of experience with Wyatt systems. We have employed them to characterize monoclonal antibodies, antibody-drug conjugates, other recombinant proteins, and vaccines.

It was clear to us, from the growing demand for AAV analytics and the capabilities offered by the SEC-MALS system from Wyatt, that we needed to present this service to our customers.

Image Credit: ShutterShock/Kateryna Kon

Light scattering techniques can be used to replace a range of different analytical techniques. Along with concentration detectors, light scatteringcan be considered a muti-attribute method that allows the analysis of size, molecular masses of the protein and the nucleic acid component, concentration, empty/full ratio and aggregate content with one system.

Additionally, it doesnt require primer or antibody reagents and it offers better precision compared to PCR and ddPCR and increased robustness to AUC. A final benefit to highlight is the simplicity of implementing the method in a GxP environment.

Thanks to the 21 CFR Part 11 compliance offered by Wyatts products, we can now employ AF4-MALS in a GxP-compliant environment.

To include data in a regulatory dossier for a characterization study, it is always essential to have complete data integrity. Such compliance with AF4 is not only an advantage for us but is also a significant benefit for our customers.

For AAV analytics in a GxP-compliant environment, Quality Assistance can offer SEC-MALS and AF4-MALS to our customers. This new flexibility in analytical methodologies offers notable advantages, including an improved time to market, thanks to the ability to analyze several critical quality attributes with a single method.

At present, the AAV market is growing. Though it is clear that there will be a need for robust and reliable analytical techniques, it is difficult to say where such developments will stop and what the future will look like. Many developments can currently be seen in analytical techniques, both in the physical/chemical part and the biological methods.

Image Credit: ShutterShock/Kateryna Kon

Further developments will invariably take place. It is difficult to say precisely which changes will be applied in a compliant environment. Still, it is likely that the analytical market for AAVs will continue to expand.

Wyattsystems are not only for AAV-based products but also for recombinant proteins such as monoclonal antibodies, antibody-drug conjugates, and vaccines such as polysaccharide vaccines. Another use is the development of a range of applications for mRNA-based therapies.

Image Credit: Wyatt Technology

Wyattsystems are versatile and flexible and can rapidly analyze and produce results from different samples in a range of formulations. Finally, all our work is performed in a compliant environment, which is vital for our customers.

The Emerging Field of Gene Therapy with Adeno-Associated Viruses - Wyatt and Quality AssistancePlay

Arnaud Delobel is the R&D and Innovation Director at Quality Assistance. He graduated from ECPM Strasbourg and specialized in mass spectrometry of proteins and peptides during his Ph.D. at the CNRS in Gif-sur-Yvette (France). After a post-doc in Lige (Belgium) focused on proteomics, he joined Quality Assistance in 2006 and held several positions in the company, eventually becoming R&D and Innovation Director in 2016.

As the head of the R&D Department, he supervises internal R&D projects, aiming at implementing new analytical technologies that Quality Assistance will ultimately provide to their customers. He also oversees a team of Product Experts responsible for identifying market needs and interpreting and translating this intelligence into developing new R&D services.

This information has been sourced, reviewed and adapted from materials provided by Wyatt Technology.

For more information on this source, please visit Wyatt Technology.

Disclaimer: The views expressed here are those of the interviewee and do not necessarily represent the views of AZoM.com Limited (T/A) AZoNetwork, the owner and operator of this website. This disclaimer forms part of the Terms and Conditions of use of this website.

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The Emerging Field of Gene Therapy with Adeno-Associated Viruses - AZoM

As activity in this space grows, so do the hurdles in moving these products forward.

Cell and gene therapy (CGT)its risks and promisesare succinctly summarized in this description of clinical trial number NCT01129544, a Phase I/II study in children born with X-linked severe combined immunodeficiency (SCID-X1), an inherited, rare, and life-threatening disease. The eight-person trial, which began in May 2010, continues today. The following paragraph has been edited.1

Gene transfer is still research for two reasons. One, not enough children have been studied to tell if the procedure is consistently successful. [And] we are still learning about its side effects and doing gene transfer safely. In previous trials, five children developed gene transfer-related leukemia; four are in remission; one died.

If the above information has stifled the research communitys scientific curiosity about CGT, it is not evident. Evidence from numerous sourcesClinicalTrials.gov, the Alliance for Regenerative Medicine (ARM), FDAare chock-a-block with studies, trials, and figures showing these therapies popularity. In the second quarter of 2022, 3,633 such treatments were in development, up from 1,745 in May 2021. The vast majority are in the preclinical stage.2,3

Some sources are revealing more.

Most indicate that academics now have a remarkable presence in the CGT development space, including sponsorship. Last year, for the first time, ARM included sponsorship figures in its twice-annual industry report.4 Academic- and government-sponsored trials far exceeded industry for sponsored trials in CGT. Stephen Majors, senior director for public affairs, ARM, says the alliance knew of academias presence for the past few years, but only was able to get data this year from its partner, Global Data.

Less reliable, but still noteworthy, are data from ClinicalTrials.gov: for active Phase I trials, industry has 89; others, which covers academia and government, have 50. Industry enrollment for Phase I is 172; others, 116.Phase III is one for others, eight for industry.

A little disruption in pharmas corner of the world? It seems that way. While basic bench to preclinical to clinical trial has long been the traditional route to FDA approvaland no one interviewed for this article suggested a reroutewhat it does imply is that pharma members have some competition from the spin-offs and academic biotechs that historically they have absorbed.

There are suspected trends that we are watching, says Majors.As to whether academias presence in this spot can be called a trend depends on ones definition of what a trend is. The Centers for Disease Control and Prevention (CDC) considers changes over a number years to determine a trend; financial investment firms typically evaluate over a two-year period.Considering that CGT companies raised $23.1 billion in 2021, 16% more than 2020,3 the answer to the above question could be, maybe.

The CGT space is still immature, according to Mike Rea, founder of Protodigm, a self-described exploratory research organization that partners with biopharma clients on alternative development and commercial solutions. Physicians need time to be comfortable with these therapies, notes Rea, so they may not be used on a regular basis.

For example, physicians have to understand how to deliver the gene, agrees cardiologist Arthur M. Feldman, MD, PhD, whose lab worked on a heart failure-related mutation in BAG3 for decades.

Last month, the company he founded, Renovacor, agreed to be acquired by Rocket Pharmaceuticals.5 We are asking physicians to do something they never did before and to understand a very different set of information, including risk/benefit discussions that they didnt learn about in medical school, he says. Feldman is a Laura H. Carnell Professor of Medicine, Division of Cardiology, and a member of the Center for Neurovirology and Gene Editing at the Lewis Katz School of Medicine at Temple University.

Chris Learn, Parexels vice president of cell and gene therapy, is unequivocal regarding academias increased presence in the drug development space focused around these treatments. He cites MD Anderson and Moffitt Cancer Center as two institutions that are sponsoring their own trials. The lines are really blurring here, he tells Applied Clinical Trials. It is indisputable.

The following is a look at how academia is showing up in various reports.

In its 2022 report4, ARM separated sponsorship, type of therapygene, cell-based, and tissue engineeringand trial phase. What these data show are industry far exceeding academic and government sponsored trials for gene therapy, while for cell therapy alone, the reverse is true: 656 cell therapy trials for academic and government, and 424 for industry. For gene therapy, there are 84 for the academics and government, and 222 for industry. In a later report, ARM found non-industry trials dropped.

Pharma Intelligences Pharma R&D Annual Review does not break down trials by their sponsors. It does, however, break down whats in the pipeline in various categories, including by the number of therapies per company, and by disease type.6 In numbers captured prior to March 2020, the analysis reported 1,849 companies with asingle drug in its pipeline, up from 1,633 in 2019, comprising more than half of all drug companies. As for types of therapies, gene therapy was in third place, the same spot it occupied in 2019. (Cancer-related therapies occupy the top spots.) Overall, biotech therapies in the pipeline increased by 13.2% in 2020 over 20196,135 vs. 5,422. Cellular therapy, the field in which academia is dominating, rose to 14th place, up from 33.

In 1982, Feldman was a resident in the cardiac care unit at the Johns Hopkins Hospital in Baltimore when he took care of a 22-year-old woman, a native Pennsylvanian, who was dying of heart failure. Sadly, we didnt have drugs with which to treat her, he recalls. Feldmans involvement with the case and the womans family led to his career as a cardiologist, he says. Twenty years later in Philadelphia, he was asked to see a heart-failure patient in consult, who turned out to be the aunt of the younger woman. It would take almost another 10 years until the technology became available to identify the genomic anomaly in this family. Here, a genetic variant that is produced by one of two alleles causes the protein product to be unstable. The result: the cell removes it, so the person with the variant has just half the amount of required protein.

BAG3 is an interesting protein that is found in the heart, the skeletal muscles, and the nervous system, including the brain. Its function is to help remove degraded and misfolded proteins, stop apoptosis or programmed cell death, and maintain the structure of the skeletal muscles. A missing allele isnt the only genetic cause for heart failure, Feldman said. Other patients, while having the correct amount of DNA, have a point mutationa single amino acidin half of the produced DNA. That single letter is the wrong amino acid in the specific site in the protein.

Around this time, Kamel Khalili, PhD, Laura H. Carnell Professor, and chair of the department of microbiology, immunology, and inflammation; director of the Center for Neurovirology and Gene Editing; and director of the Comprehensive NeuroAIDS Center, Lewis Katz School of Medicine, Temple University, had created a method by which he could excise the HIV virus from patients using the new technique of CRISPR-Cas9.

Khalili believes that BAG3 may be involved in the pathogenesis of HIV-1 in brain diseases and protein quality control caused by viral infection as well as several other disorders, including Alzheimers disease and dementia. BAG3 changes the homeostasis of the cell, he says. The only solution is to fix the cell. Khalili has used CRISPR technology to excise the viral genome in both small and large investigational animals and has recently started a Phase I trial to test the safety of the new gene-editing treatment. Khalili, too, started a company, but Temple holds the license. In the case of Renovacor, it was granted the license by Temple.

As a scientist, when you are doing something in biomed research, [the] goal is to translate bench work to the clinic for [the] wellness of people. We are doing long hours and long days because we want to help. We are trying to see if discovery can help people, says Khalili. I know my limit, I stop at business aspects. My interest is to discover research which can help populations.

Was Feldman happy with his business experience? As a company gets bigger, others join the team who fulfill other roles, like acquiring funding or developing the actual product, he says. Releasing the control reins are difficult. But if it speeds up the timeline to get an approved product into the clinic, then its all worth it, he adds.

Researchers such as Feldman and Khalili, says Kaspar Mossman, PhD, director of communications and marketing at QB3, a University of California biotech accelerator, are normally not deeply interested in business. He notes the new flagship space in UC Berkeley called Bakar Lab. So far, it has 25 companies, one-third from university labs. They collaborate, they share equipment, [at times] they merge, Mossman tells Applied Clinical Trials.

And, he adds, Academics tend to be very smart individuals. The more time they spend in business, they learn stuff and become serial founders, says Mossman. They are honest about not wanting to be a CEO.

In terms of business, the academics employers are also pretty smart. The huge bugaboo with CGT commercialization is the manufacturing processthe need for an apheresis unit, ultra-cold storage, and regulated cell processing facilities.

Some institutions are building their own manufacturing facilities to more easily meet the increasingly complicated standards pertaining to regenerative medicine production. Harvard, MD Anderson, Moffitt, the University of Pennsylvania, and the University Hospital of Liege in Belgium8 all have or are planning to build their own facilities.

As for how academias presence impacts the traditional pharma space, those interviewed cited pros and cons. More research is better, more companies vying for venture capital funding is not. But more trials mean more competition among similar therapies, which, says Majors, is a good thing.

We need experimentation, adds Rea. If left to pharma, he says, the research wouldnt happen. Smaller biotechs are taking the risk. Over the last 10 years, Rea believes pharma has been slow in the risk-taking department. Once upon a time, pharma didnt have many competitors. Now, with many numerous smaller companies with viable assets, willing to accept a smaller net profit, the competition is creating some angst. Pharma cant project everyones movement, says Rea. The gene/cell therapy landscape [for products] is huge.

Likely adding to the angst: Those smaller biotechs are getting financial help. Between April 4, 2021, and June 24, 2021, of 23 start-up financing deals, 19 involved academics.2

Learns viewpoint is different. He says there are too many players out there, and while large pharma may be averse to risk, I really do believe what we are witnessing are simply market forces that have played into this. There is so much cash coming in, he continues, that people can be blinded by the pitfalls. The CGT area, he adds, is bloated and he says the industry needs an overall strategy.

Learn doesnt think that academias presence in the CGT space is a flash in the proverbial pan. The enthusiasm to find cures is real, and some research institutions have the endowments to see the trials through. I think it is just the beginning, says Learn. Academia will put their futures in front of them. Why put all your sweat equity into it and not have any fiduciary benefit of the approved product?

In Pharma Intelligences 2020 Pharma R&D Review, its author questioned the wisdom of so many drugs, overall, in the pipeline4,001 added in 2018 and 4,730 added in 2019, for a total of 17,737 drug candidates. [A]re the industrys eyes getting too big for its belly? Unless it can continue to provide [approved therapies] then a certain degree of control in the pipeline might be advisable, the report stated.6

And now to costs. While no one doubts these cures change lives, the question of access persists. FDAs approval of Bluebird Bios second therapy this year, branded as Skysona, for early but active cerebral adrenoleukodystrophy, is expected to cost $3 million. Learn doubts that payers are jumping up and down to get Skysona on their formularies.

Its still a fairly dicey business proposition for companies to invest in this field, Steven Pearson, MD, president of the Institute for Clinical and Economic Review (ICER), said recently.8Theres still a risk that next-generation therapies will not flourish even in developed countries health systems, he added.

One positive development in the US, however, occurred late last month when Congress reauthorized the Prescription Drug User Fee Act (PDUFA) for the next five years, 2023-2027. The action maintained FDAs authority to collect fees from manufacturers and keep and recruit agency staff to review the increased number of CGT applications. Majors says most of FDAs review of CGT products involves scalability and consistent reproducibility in the manufacturing process, which, of course, means traveling.

According to a Senate press release9, FDA is seeking to hire at least 320 new staff members. In a statement, Pharmaceutical Research and Manufacturers of America (PhRMA) said a modern regulatory framework supported by PDUFA helps ensure patients have timely access to lifesaving medicines.

PDUFA reauthorization aside, there is little argument that the field of CGT, from research and drug discovery through commercialization, is advancing rapidly. In turn, so are the unique operational and manufacturing challenges that these therapies present. This reality may thin the currently crowded playing field in CGT going forward, with those sponsors and partners best prepared to deliver on the numerous touchpoints required separating from the pack.

Christine Bahls, Freelance Writer for Medical, Clinical Trials, and Pharma Information

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The Risk-Reward Proposition for CGT Clinical Trials - Applied Clinical Trials Online

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Key Companies Covered

In this section of the report, the researchers have done a comprehensive analysis of the prominent players operating and the strategies they are focusing on to combat the intense competition. Company profiles and market share analyses of the prominent players are also provided in this section. Additionally, the specialists have done an all-encompassing analysis of each player. They have also provided reliable revenue, market share, and rank data of the companies for the period 2022-2030. With the assistance of this report, key players, stakeholders, and other participants will be able to stay abreast of the recent and upcoming developments in the business, further enabling them to make efficient choices. Mentioned below are the prime players taken into account in this research report:

3P Biopharmaceuticals, ABL, Inc., AGC Biologics, Advent BioServices Ltd., Akron Biotech, Aldevron, Anemocyte S.r.l

Cell and Gene Therapy Manufacturing Market Segments

This report has explored the key segments: by Type and by Application. The lucrativeness and growth potential have been looked into by the industry experts in this report. This report also provides revenue forecast data by type and by application segments based on value for the period 2022-2030

Market Segments by Type:

Pharmaceutical and Biotechnology , Academic and Research Institutes, Other

Market Segments by Application:

Clinical Manufacturing, Commercial Manufacturing

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Methodology :

Market research fills the gap between goods or services and their target market or client. Market research insights assist a manufacturer or service provider in developing a product or service plan that satisfies the specific needs of the target market.

Primary Market Research:

Primary market research is a type of study that involves acquiring information from target industries, either through a third party or directly from customers.

Quantitative Market Research:

Quantitative market research is a method of gathering data from the target market and consumers that can be quantified.

Qualitative Market Research:

Qualitative market research is a means of gathering qualitative data from target markets via the use of instruments and procedures such as focus groups and interviews.

Secondary Market Research:

Secondary market research is an investigative strategy in which a firm relies on publicly available data to learn more about its customers and target markets.

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For the global version, a list of below countries by region can be added as part of customization at minimum cost:

Each of the regional market segments is analyzed and studied in the sense of the major regional spectrum of market reach in the market report. The study also provides a comprehensive overview of key insights, such as import, export, development, demand, and consumption

North America (United States, Canada, and Mexico)Europe (Germany, France, UK, Russia, and Italy)Asia-Pacific (China, Japan, Korea, India, and Southeast Asia) South America (Brazil, Argentina, Colombia, etc.)The Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

The study of the market includes

Explanation of consumer preferences, trends, opportunities, and factors impacting storage technologies, as well as an analysis of current industry trends. Market share study by end-user, service, application, and region Investments in R&D and demonstration projects are covered, as well as a full overview of the market. Assessment of regulatory trends and incentives, regional economic development rate, implementation timeline, and environmental constraints

Why You Should Buy This Report:

The report analyzes regional growth trends and future opportunities. Detailed analysis of each segment provides relevant information. The data collected in the report is investigated and verified by analysts. This report provides realistic information on supply, demand, and future forecasts.

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Cell and Gene Therapy Manufacturing Market Trends, Opportunities, and Breakthrough Point During (2022-2030)- 3P Biopharmaceuticals, ABL, Inc., AGC...

DUBLIN--(BUSINESS WIRE)--The "Biopharmaceutical Contract Manufacturing Organization (BCMO) Market, 2022" report has been added to ResearchAndMarkets.com's offering.

This report, Biopharmaceutical Contract Manufacturing Organization (BCMO) Market, 2022, describes and discusses the global contract manufacturing markets, specifically focusing on the production of biotech (large molecule) products including extensive coverage of the biotechnology segment of the global BCMO industry.

Sales estimates are provided by segment and region, expressed in current dollars. Estimates are provided for the historic 2016 to 2021 period and forecasts are provided through 2026.

Further, this report examines third party manufacturing of potential and commercialized prescription drug products throughout the world. Potential drug products are those in clinical (Phase I - Phase III) testing prior to regulatory approval and require relatively small amounts of product for these evaluations; commercialized drugs are those which have received regulatory approval and have been introduced to mass markets. In some cases, drugs have received regulatory approval in selected regions (such as the EU, but not in others such as the US).

The report contains the following market information:

Key Topics Covered:

Chapter 1: Executive Summary

Chapter 2: Overview

Chapter 3: The Contract Manufacturing Industry

Chapter 4: Total Global BCMO Market

Chapter 5: The BCMO Market by Region

Chapter 6: Pharmaceutical & Biotech Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/664983

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World Biopharmaceutical Contract Manufacturing Organization (BCMO) Market Research Report 2022: Focus on Growth Factors - Biotechnology, Biosimilars,...

Covina, Oct. 11, 2022 (GLOBE NEWSWIRE) -- Genomics is the study of all of persons gene. Genomics play role in health and disease. Genomics are widely used in cancer care treatment for diagnosing and treating cancer disease. Structural Genomics and Functional Genomics are two types of Genomics.Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics are variety of applications in genomics. Metagenomics has become the important application in genomics. The newer technique genome editing is used in gene therapy. Genome editing help to introduce gene-editing tools which can able to change existing DNA in cell. Genomics are used in drug discovery due to their properties like high-throughput sequencing & characterization of expressed human genes. Genomics has allowed effective preventive measures, change in drug research strategy and development process in drug discovery due to knowledge about human genes and their functions. A complete human genome contains about 3 billion base pairs of DNA. Pharmacogenomics is the study of genes and their functions to develop safe medications which are effective and can be prescribed based on persons genetic makeup. Pharmacogenomics choose the drug and drug doses that are effective for that particular person by using genetic information about that person. Pharmacogenomics helps in improving patient safety, health care costs and drug efficiency. Single nucleotide variant (SNV) panels are used in pharmacogenetics. Genomics helps to reveal the abnormalities in genes which has drived the development and growth of different types of cancer.Study of cancer genome has improved in understanding the biology of cancer which has enabled to discover new methods for diagnosing & treating the disease. The importance of Genomics in cancer care has provided to discover new drug development and effective treatment in diagnosing and treating the disease which has driven positive impact on target market growth.

The reportGlobal Genomics in Cancer Care Market, By Type (Structural Genomics, Functional Genomics), By Application (Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics, and Others), By End-User (Research Institute, Hospitals, Academic Research Institutes, Diagnostic Centers, and Others) andBy Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2032

Key Highlights:

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Analyst View:

Increase in cancer disease, rising emergence of clinical relievance in genomic medicine, recent advancement in genomics, newly developed technology like next-generation sequencing has given rise in use ofGenomics in Cancer Care. Wide variety of applications in Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics has fueled the target market growth. Rising awareness in individual who are pertaining to cancer genomics, rapid growth in biotechnology industries, research institutes, diagnostic centers is expected to have positive impact on Genomics in Cancer Care market. Importance of Genomics in cancer care has enabled to provide effective treatment, new drug development, diagnosing and treating disease which has enhanced the target market growth.As a result, market competition is intensifying, and both big international corporations and start-ups are vying to establish position in the market.

Browse 60 market data tables* and 35figures* through 140 slides and in-depth TOC onGlobal Genomics in Cancer Care Market, By Type (Structural Genomics, Functional Genomics), By Application (Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics, and Others), By End-User (Research Institute, Hospitals, Academic Research Institutes, Diagnostic Centers, and Others) andBy Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2032

To know the upcoming trends and insights prevalent in this market, click the link below:

https://www.prophecymarketinsights.com/market_insight/Genomics-in-Cancer-Care-Market-4953

Key Market Insights from the report:

GlobalGenomics in Cancer CareMarketaccounted for US$ 16.1 Bn in 2022 and is estimated to be US$ 72.61 Bn by 2032 and is anticipated to register a CAGR of 16.3%.TheGlobalGenomics in Cancer CareMarketis segmented based on Type, Application, End-User and Region.

Competitive Landscape & their strategies ofGlobalGenomics in Cancer Care Market:

The prominent players operating in theGlobalGenomics in Cancer CareMarketincludes,Pacific Biosciences Inc., Abbott Molecular Oxford Gene Technology, Roche Diagnostics, Bio-Rad Labs, Illumina Inc., Quest Diagnostics, Beckman Coulter Inc., Intrexon Bioinformatics Germany GmbH, Agilent Technologies, PerkinElmer, Danaher Corporation, Cancer Genetics Inc., Thermo Fisher Scientific Inc., and others.

The market provides detailed information regarding the industrial base, productivity, strengths, manufacturers, and recent trends which will help companies enlarge the businesses and promote financial growth. Furthermore, the report exhibits dynamic factors including segments, sub-segments, regional marketplaces, competition, dominant key players, and market forecasts. In addition, the market includes recent collaborations, mergers, acquisitions, and partnerships along with regulatory frameworks across different regions impacting the market trajectory. Recent technological advances and innovations influencing the global market are included in the report.

Scope of the Report:

About Prophecy Market Insights

Prophecy Market Insights is specialized market research, analytics, marketing/business strategy, and solutions that offers strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high-value opportunities in the target business area. We also help our clients to address business challenges and provide the best possible solutions to overcome them and transform their business.

Some Important Points Answered in this Market Report Are Given Below:

Browse for Related Reports:

1.Single-Cell Genome Sequencing Market, By Type (Reagents and Instruments), By Technology (NGS, PCR, qPCR, MDA, and Microarray), By Application (Circulating Cells, Genomic Variation, Cell Differentiation, Subpopulation Characterization, and Others), By End-User (Forensic Labs, Cell banks and IVF centers, Academic & Research Laboratories, Biotechnology & biopharmaceutical companies, and Hospitals and diagnostic laboratories) and By Region (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa) - Trends, Analysis and Forecast till 2029

2.Global Genomics Market By Product and Services (Consumables, Instruments/Systems, and Services), By Technology (Microarray, Purification, PCR, Sequencing, Nucleic Acid Extraction, and Other Technologies (Gene Editing, Gene Expression, Genotyping, and among others)), By Process (Library Preparation, Sequencing, and Data Analysis), By Application (Diagnostics, Precision Medicine, Agriculture, Drug Discovery & Development, Animal Research, and Other applications (Biofuels, Coal Mines, Marine Research, and Among Others)), By End User (Academic &Government Institutes, Research Centers, Hospitals & Clinics, Pharmaceutical & Biotechnology Companies, and Other End Users (Agri-genomics organizations, NGOs, among others)), and By Region (North America, Europe, Asia Pacific, Middle East, and Africa) - Trends, Analysis and Forecast till 2029

3.Global Lab-On-a-Chip Market, By Product Type (Instruments, Reagents and Consumables, Software, and Services), By Application (Genomics and Proteomics, Diagnostics, Drug Discovery, Others), By End-User (Hospitals, Academic and Research Institute, Diagnostic Lab, Homecare settings, and Others) and By Geography (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa)- Trends, Analysis and Forecast till 2029

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Genomics in Cancer Care Market is estimated to be US$ 72.61 billion by 2032 with a CAGR of 16.3% during the forecast period 2032 - By PMI -...

Renewed National Institute for Health and Care Research Birmingham Biomedical Research Centre continue funds for developments around inflammatory diseases

Published 14 October 2022

Local people with cancer and heart disease are amongst those set to benefit from a major injection of research funding which will develop new diagnostic tools and treatments for those with cancer, liver and heart disease, and many more illnesses.

The NIHR Birmingham Biomedical Research Centre has today been granted more than 30 million of funding from the National Institute for Health and Care Research, a major funder of global health research and training, to support world-leading research into inflammation and the myriad of diseases and health issues that it can cause.

The centre unites leading NHS providers led by the University Hospitals Birmingham NHS Foundation Trust and academic institutions led by the University of Birmingham. The partnership sees eight organisations working closely with charities and businesses to support research into inflammation which causes or worsens many common long-term illnesses including arthritis, liver disease and cancer.

The announcement sees the NIHR Birmingham BRC increase its funding almost 3-fold and will enable researchers to focus on eight areas of illness including heart disease, womens health, and common complications from inflammation. It will also enable researchers to consider new tests and biomarkers for disease, health technologies including stem cells and gene therapy, patient experiences and data science.

The funding will allow us to make a step-change in our work tackling different forms of cancer, trialling new drugs for liver disease, and dealing with antimicrobial resistance.Professor Philip Newsome

Professor Phil Newsome, Director of Research and Knowledge Transfer at the University of Birminghams College of Medical and Dental Sciences and Director of the NIHR Birmingham Biomedical Research Centre said:

Inflammation plays a central role in many health conditions, with millions of people in the UK alone experiencing inflammatory diseases such as arthritis and bronchitis.

The significant increase in funding for the NIHR Birmingham Biomedical Research Centre will enable us to provide an outstanding environment for world-leading clinical research. The funding will allow us to make a step-change in our work tackling different forms of cancer, trialling new drugs for liver disease, and dealing with antimicrobial resistance.

Patients will benefit from the increased funding for the NIHR Birmingham BRC through collaborative research that has seen nearly 1,000 clinical trials and informed UK clinical guidelines.

Researchers will look at eight themes to continue to understand and help patients manage inflammation-based diseases including cancer, arthritis, and liver disease. The investment of the NIHR funding in biomedical research will enable clinicians, researchers, patients and supporters to find new treatments such as the development of new immunotherapies, which are types of cancer treatments to support the body to fight cancer.

When Joy needed a liver transplant, the team at University Hospitals Birmingham involved in her care made her aware about an opportunity to take part in a trial through the BRC to better understand sarcopaenia.

In many chronic inflammatory diseases, including liver disease, loss of muscle mass and strength (sarcopaenia) occurs. Importantly sarcopaenia contributes to poor patient quality of life, reduced ability to cope with challenges such as surgery or infection and higher risk of death. The NIHR Birmingham BRC has been investigating inflammatory sarcopaenia to find the best treatments and support for patients who experience inflammation-related muscle loss.

Joy said: It was interesting to be made aware of muscle loss and to be encouraged to do tests [to assess sarcopaenia]. Following the two-day assessment, I was encouraged to increase protein in my diet because of muscle loss and the trial reinforced how important that was. I think the trial made me feel like I was doing something positive at a time when some things in my life were dampening down.

After the transplant, one of the first people I saw in hospital was a member of the research team. She had heard I had had the op and made a point of dropping by. We had a lovely chat. As a beneficiary of liver transplant and the improving outcomes for liver transplant patients, I am heartily in favour of research in this as well as other medical fields.

Tim Jones, Chief Innovation Officer, at University Hospitals Birmingham NHS Foundation Trust said:

We are delighted with the award to the NIHR Birmingham BRC which builds on our successful track record of joint working in Inflammatory disease, the award will significantly support the acceleration of new discoveries for the benefit of our patients.

Researchers based at the NIHR Birmingham BRC will also support a major improvement in how data and digital healthcare can improve patient outcomes.

The Data, Diagnostics and Decision Tools theme will see the development of new infrastructure and innovation to tackle major challenges in the use and interpretation of data in biomedical research. Bringing together expertise across a range of disciplines including clinical trials, health informatics, and artificial intelligence, increased funding will see improvements in the way data is held and used to uphold the highest levels of research integrity.

The expanded funding will also enable the NIHR Birmingham BRC to invest in research excellence and create additional capacity, to collaboratively focus on key wider areas of clinical practice including the development of new tests and biomarkers, and next generation therapies such as stem cell and gene therapy.

Mark Maybury joined the Birmingham Rheumatology Research Group in 2018, supported by the NIHR-funded Birmingham Biomedical Research Centre. Coming from a clinical role as a physiotherapist and musculoskeletal sonographer, Mark has been working with academics to support research trials for rheumatoid arthritis drugs and has been involved in pioneering work in the use of ultrasound-guided synovial joint biopsies.

Mark said: One of the many reasons that attracted me to move into this research post at this institution was the opportunity to learn and during my time at the Birmingham BRC I have been given the opportunity for personal study and involvement in research. But as well as learning myself, Ive also had the opportunity to pass on my knowledge - training research fellows in diagnostic ultrasound and to develop new instrumentation for ultrasound guided synovial biopsy. The results of the studies I have worked on will help influence the treatment of hundreds of thousands of patients, not only in the UK but worldwide, which is more patients than I ever could ever help as a clinician. I find that a very sobering thought."

The investment from NIHR is hugely important for researchers working across the BRC partner institutions, to continue to tackle some of the critical health themes that affect our region.Professor David Adams

Professor David Adams, Pro-vice Chancellor and Head of the College of Medical and Dental Sciences at the University of Birmingham, and director of the previous NIHR Birmingham BRC said:

The investment from NIHR is hugely important for researchers working across the BRC partner institutions, to continue to tackle some of the critical health themes that affect our region.

"The funding will allow us to bring together teams that can use an in depth understanding of disease processes to deliver new therapies and diagnostic tests for a range of chronic inflammatory diseases for which we currently have few effective treatments.

The NIHR Birmingham BRC is among 20 centres across England that have been awarded a combined 790 million by the National Institute for Health and Care Research, to translate scientific discoveries into new treatments, diagnostic tests and medical technologies for patients.

NIHR Biomedical Research Centres are partnerships between healthcare professionals and academics in the countrys leading NHS trusts and universities. The centres, part of NIHRs research infrastructure, receive substantial levels of sustained funding to attract the best scientists and create an environment where experimental medicine can thrive.

The Birmingham Biomedical Research Centre is made up of the following partners:

Professor Lucy Chappell, Chief Executive of the NIHR, said:

Research by NIHR Biomedical Research Centres has led to a number of ground-breaking new treatments, such as new gene therapies for haemophilia and motor neurone disease, the world-first treatment for CreutzfeldtJakob disease, a nose-drop vaccine for whooping cough, and the first UK-wide study into the long-term impact of COVID-19.

This latest round of funding recognises the strength of expertise underpinning health and care research across the country and gives our nations best researchers more opportunities to develop innovative new treatments for patients.

For media enquiries for the University of Birmingham, please contact Tim Mayo, Press Office, University of Birmingham, tel: +44 (0)7920 405040: email: t.mayo@bham.ac.uk

The mission of the National Institute for Health and Care Research (NIHR) is to improve the health and wealth of the nation through research. We do this by:

NIHR is funded by the Department of Health and Social Care. Its work in low and middle-income countries is principally funded through UK Aid from the UK government.

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New 30M research injection to improve treatment of inflammatory diseases - University of Birmingham

Naoto T. Ueno

Naoto T. Ueno has been selected to be the next director of the University of Hawaii Cancer Center. Uenos appointment is pending the UH Board of Regents approval at the boards October 20 meeting. His appointment would be effective December 12, 2022.

Ueno is currently the executive director of the Inflammatory Breast Cancer Research Program at the University of Texas MD Anderson Cancer Center, where he is also a tenured professor of medicine.

He has held various faculty and administrative positions there since 1996. Ueno has 15 years of leadership experience, and nearly 30 years of experience in research and education, particularly in stem cell transplant, gene therapy, targeted therapy and immunotherapy.

His research focus is inflammatory breast cancer (IBC)the most lethal and aggressive form of breast cancer with a high metastasis (spread) rate. Working with his team members, he created a comprehensive research program and clinic devoted to IBC, which, under his leadership, emerged as the worlds largest and most renowned for rare breast cancer.

Ueno is also widely regarded for his preclinical development and research efforts that translated into clinical trials. These valuable outputs will be of great benefit to the cancer centers clinical trials and community building efforts.

Dr. Ueno possesses the leadership skills, background and experience to lead the UH Cancer Center for years to come, I am confident he will continue to advance the UH Cancer Center and their mission to reduce the burden of cancer in communities throughout Hawaii, said UH Mnoa Provost Michael Bruno.

Ueno said his mission as director is to ensure that every patient with cancer lives to the fullest extent. He wishes to prevent cancer in Hawaii and the Pacific through patient-centered cancer strategies, high-quality cancer care, research, prevention and education. In his previous roles, he helped build a team culture reflecting diversity and psychological safety.

I am honored and excited for this tremendous opportunity to work with a new team and to build collaborative partnerships inside and outside of the UH Cancer Center, said Ueno. Being raised in both Asia and the U.S., I have a long history of interest in Hawaiis diverse culture and demographics. I truly believe the UH Cancer Center has a unique opportunity to build a world-class clinical and translational program that serves the community to reduce the suffering of cancer patients.

As a cancer survivor, Ueno brings more depth in his role as director, rendering the ability to empathize with cancer patients. He has also published two books about empowering patients in cancer care.

Dr. Uenos leadership experience, coupled with his ability to share common ground with cancer patients, make him uniquely qualified to improve cancer patient care in Hawaii and the Pacific, said Bruno.

Ueno earned his MD from Wakayama Medical College in Japan. He went on to earn his PhD in cancer biology from The University of Texas Graduate School of Biomedical Sciences. He received postgraduate training through internships and fellowships with the United States Naval Hospital in Yokosuka, Kanagawa Japan, Montefiore University Hospital (internal medicine), the University of Pittsburgh Medical Center (internal medicine) and The University of Texas MD Anderson Cancer Center (medical oncology and stem cell transplantation).

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Naoto T. Ueno announced as new UH Cancer Center director | University of Hawaii System News - University of Hawaii

A biotechnology company launched Wednesday by life sciences venture firm ATP is the latest startup to debut with a new twist on genetic editing.

With $50 million in funding, Boston-based Ascidian Therapeutics claims its RNA exon editing approach could match the durability of gene therapy while avoiding some of the risks that come with editing DNA.

Its platform is designed to correct for mutations in exons the regions of DNA that contain information needed to make proteins. Ascidian aims to do this by replacing mutated exons with functional RNA copies as DNA is being converted into its chemical cousin.

The company will first target a genetic eye condition called Stargardt disease, which is the most common form of inherited macular degeneration and results in vision loss.

According to Ascidian, its technology can fix genetic errors that other editing approaches cant, and can be applied to widely varied genes. Its lead program can replace more than 20 exons at a time, said Romesh Subramanian, Ascidians CEO.

We are changing chapters in a book rather than whiting-out one letter at a time, Subramanian, said in an interview with BioPharma Dive. Subramanian came to Ascidian from Dyne Therapeutics, a biotech he founded and led as CEO. He previously founded RNA specialist Translate Bio, which was bought by Sanofi last year.

Subramanian claims that Ascidians approach, by focusing on RNA, maintains genome integrity and thereby sidesteps concerns around off-target edits. His company also doesnt rely on foreign enzymes to work, potentially easing immunogenicity risks, he added.

Along with Stargardt disease, Ascidian is looking at other eye conditions, neurological disorders and rare diseases. Subramanian declined to disclose how many drug research programs Ascidian plans to roll out.

Ascidians name is derived from a class of ocean-dwelling invertebrate creatures, which are sometimes known as sea squirts. These creatures use RNA trans-splicing to alter the RNA messengers used by their cells, a process that Ascidian plans to leverage to rewrite RNA for treating disease.

Ascidian is not ATPs first foray into genetic medicine. Last year, Ascidian co-founder and ATP venture partner Michael Ehlers, a former Biogen executive,launched a startup called Intergalactic Therapeuticsthat focuses on non-viral gene therapy. ATP has also built a company called Replicate, which is developing another kind of RNA medicine.

We think the RNA space is a big way of manipulating biology and treating disease across the board, and this approach we've taken to Ascidian defines a new class of RNA therapeutics, Ehlers said.

The company expects to spend the rest of 2022 and 2023 on pre-clinical studies for its lead program, along with developing proof of concept for other candidates targeting neurological and neuromuscular diseases.

Gene editing research was catalyzed by the discovery of CRISPR, which has now been extended and adapted to support several different gene editing technologies. But biotech companies are also exploring RNA editing, which in part appeals to scientists because it doesnt change the underlying DNA.

It has drawn in larger drugmakers, too: Roche and Eli Lilly have recently formed partnerships with Shape Therapeutics and ProQR Therapeutics, respectively, to develop treatments for a wide variety of diseases.

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Ascidian starts up with $50M and a twist on RNA editing - BioPharma Dive

ReportLinker

The Global Viral Vectors Market size was estimated at USD 1,291. 23 million in 2021 and expected to reach USD 1,464. 47 million in 2022, and is projected to grow at a CAGR 13. 67% to reach USD 2,785.

New York, Oct. 14, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Viral Vectors Market Research Report by Type, Disease, Application, End User, Region - Global Forecast to 2027 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p06342341/?utm_source=GNW 63 million by 2027.

Market Statistics:The report provides market sizing and forecast across 7 major currencies - USD, EUR, JPY, GBP, AUD, CAD, and CHF. It helps organization leaders make better decisions when currency exchange data is readily available. In this report, the years 2018 and 2020 are considered as historical years, 2021 as the base year, 2022 as the estimated year, and years from 2023 to 2027 are considered as the forecast period.

Market Segmentation & Coverage:This research report categorizes the Viral Vectors to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Type, the market was studied across Adeno-associated Viral Vectors, Adenoviral Vectors, and Retroviral Vectors.

Based on Disease, the market was studied across Cancers, Genetic Disorders, and Infectious Diseases.

Based on Application, the market was studied across Gene Therapy and Vaccinology.

Based on End User, the market was studied across Pharmaceutical & Biopharmaceutical Companies and Research Institutes.

Based on Region, the market was studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, Thailand, and Vietnam. The Europe, Middle East & Africa is further studied across Denmark, Egypt, Finland, France, Germany, Israel, Italy, Netherlands, Nigeria, Norway, Poland, Qatar, Russia, Saudi Arabia, South Africa, Spain, Sweden, Switzerland, Turkey, United Arab Emirates, and United Kingdom.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, and the long-term effects are projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlying COVID-19 issues and potential paths forward. The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Cumulative Impact of 2022 Russia Ukraine Conflict:We continuously monitor and update reports on political and economic uncertainty due to the Russian invasion of Ukraine. Negative impacts are significantly foreseen globally, especially across Eastern Europe, European Union, Eastern & Central Asia, and the United States. This contention has severely affected lives and livelihoods and represents far-reaching disruptions in trade dynamics. The potential effects of ongoing war and uncertainty in Eastern Europe are expected to have an adverse impact on the world economy, with especially long-term harsh effects on Russia.This report uncovers the impact of demand & supply, pricing variants, strategic uptake of vendors, and recommendations for Viral Vectors market considering the current update on the conflict and its global response.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Viral Vectors Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis:The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

Competitive Scenario:The Competitive Scenario provides an outlook analysis of the various business growth strategies adopted by the vendors. The news covered in this section deliver valuable thoughts at the different stage while keeping up-to-date with the business and engage stakeholders in the economic debate. The competitive scenario represents press releases or news of the companies categorized into Merger & Acquisition, Agreement, Collaboration, & Partnership, New Product Launch & Enhancement, Investment & Funding, and Award, Recognition, & Expansion. All the news collected help vendor to understand the gaps in the marketplace and competitors strength and weakness thereby, providing insights to enhance product and service.

Company Usability Profiles:The report profoundly explores the recent significant developments by the leading vendors and innovation profiles in the Global Viral Vectors Market, including ABL Inc., Batavia Biosciences B.V., BioNTech IMFS GmbH, Biovian Oy, Cell and Gene Therapy Catapult, Cevec Pharmaceuticals GmbH, Creative Biogene, FinVector Vision Therapies, Fujifilm Diosynth Biotechnologies, GeneOne Life Science, Inc., Genezen Laboratories, Lonza Group AG, Merck KGaA, Miltenyi Biotec GmbH, Novasep Inc., Sirion-Biotech GmbH, Spark Therapeutics Inc., Thermo Fisher Scientific Inc., and Wuxi AppTec Co., Ltd..

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as:1. What is the market size and forecast of the Global Viral Vectors Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Viral Vectors Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Viral Vectors Market?4. What is the competitive strategic window for opportunities in the Global Viral Vectors Market?5. What are the technology trends and regulatory frameworks in the Global Viral Vectors Market?6. What is the market share of the leading vendors in the Global Viral Vectors Market?7. What modes and strategic moves are considered suitable for entering the Global Viral Vectors Market?Read the full report: https://www.reportlinker.com/p06342341/?utm_source=GNW

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Viral Vectors Market Research Report by Type, Disease, Application, End User, Region - Global Forecast to 2027 - Cumulative Impact of COVID-19 - Yahoo...

Gene therapy is a medically-based practice that uses normalized genetics to replace genes which are either not present or abnormal for some individuals. Doctors would take the specific gene sequences that need adjustment, and then insert them into the cellular information of the patient in various ways. Most forms of gene therapy are still in the clinical research stage, but there have been stories of encouraging results.

Several inherited immune deficiencies are being treated successfully right now with gene therapy. When the blood stem cells are removed from patients, retroviruses then deliver working copies of the defective genes to the body.

For the gene therapy options which have been approved for use, there are many success stories to consider. Sebastian Misztal is one such story. He was a patient in a hemophilia gene therapy trial in 2011. After receiving the therapy, Misztal no longer experiences episodes of spontaneous bleeding.

Roughly 70% of the currently active gene therapy clinical trials are based in the United States. Europe approved their first treatment in this area in 2012. These are the pros and cons of this scientific approach to consider.

1. Gene therapy provides hope for those who may not have had any in the past.About 3% of American children are born with a genetic condition which requires gene therapy as a way to treat the issue. At this time, the diseases and disorders which are present in this population will take the life of the child before there is an opportunity to correct the condition. Birth defects are the leading cause of newborn death in the United States, with as many as 1 in 5 children suffering from them. Advances in gene therapy could help to correct these issues instead of forcing parents into a heartbreaking scenario.

2. Gene therapy could change the perspectives that people have about disease.Roughly 10% of all Americans are affected by a rare disease or condition on any given day. Approximately 33 million people are suffering from a disability that is directly attributed to their genetic profile. The promise of gene therapy is that it can reduce or eliminate the pain and discomfort that these abnormalities cause. 80% of the diseases that we know impact human health in negative ways have a genetic foundation. If we can replace the cells or chromosomes that are at-fault, then it becomes possible to offer relief.

3. Gene therapy could offer the potential of new discoveries.Our world is a better place when there is an emphasis on diversity. When we have effective gene therapy treatments that can save lives or prolong them, then we are adding strength to our existence. There will be more opportunities to research, new ideas that could lead to critical discoveries, and relationships that can lead to future generations that experience these benefits as well. There will always be a segment of society that looks at gene therapy as a way to play God. The reality of this medical treatment is that it can help people continue to live a life that they love.

4. Gene therapy could be used in different ways to improve life.Right now, the focus of gene therapy research is to provide solutions for people who are suffering from specific illnesses or diseases. When we begin to experience successes in this field, then the information we learn can apply to other treatment areas as well. Gene therapy could be useful in the treatment of infertility issues. The processes involved may help people struggling with vision or hearing issues. Even if the only thing that we can do with this science is to relieve chronic pain, that would be tremendously beneficial for the futures of many people.

5. Gene therapy does not just apply to human treatment options.When we discuss the pros and cons of gene therapy, it is essential to remember that the benefits we can experience as humans apply to other forms of life as well. The technologies we create from this research could help us to grow crops that adapt more effectively to changing climate conditions. We could use this information to correct the various genetic conditions that we know about in the animal kingdom. This data could help us to grow healthier foods, increase the shelf life of harvests, or produce more items in our overall yields.

6. Gene therapy allows us to use technology to improve the quality of life for people.Many of our medical discoveries rely on technological processes that we apply to natural items. Even some of the most critical advancements of our era, such as the development of a polio vaccine by Dr. Jonas Salk (and the work of many others) relied on the use of inactivated virus materials to create the first usable product. Gene therapy would become one of the first treatment options for doctors that was purely technological. That means our opportunity to develop new resources from it are virtually unlimited.

7. Gene therapy allows us to treat the untreatable diseases.Gene therapy is potential miracle worker when we start to look at its full potential for humanity. It offers us the opportunity to eliminate, and then prevent hereditary diseases like hemophilia and cystic fibrosis. The technologies behind this treatment option could provide us with a possible cure for heart disease. Potential medical options include cancer and AIDS cures. Even if there is a fair amount of risk involved when treating these health issues, there are a lot of patients who dont have much to lose. Gene therapy opens a door that we once thought was permanently locked.

1. Gene therapy does not have a reliable delivery method.Retrovirus delivery systems are the most common way for gene therapies to be delivered to patients. The problem with this option is that the enzyme used to encourage the transfer of genetic data can be eliminated by the immune system before it has the chance to work. There could be issues with cell division or replication that limit the effectiveness of the treatment.

When there is a noticeable change to the cell, the body might attack itself without the presence of an immunosuppressant. Until we can remove and replace genetic data with more reliability, the success stories for gene therapy will always be hit or miss.

2. Gene therapy is an expensive procedure.There are several gene therapy options which are available right now, but they come at a steep price. If you use Luxturna to treat both eyes as a way to treat blindness, then the final cost could be more than $1 million. Even the affordable options in this field start at $200,000 per treatment. Thats why many patients weight for clinical trials to begin, and then apply for a spot in one to receive the help they need. Most healthcare insurance plans will not cover the cost of these procedures because of their uncertainty.

3. Gene therapy requires ongoing treatment s to be effective.Many people have found that the benefits of their gene therapy treatments began to wear off as soon as they were no longer taking their medicine or visiting their doctor for treatments. It can be a lifelong course that someone must follow to reduce or eliminate the genetic issues that hold back their health. Unless you can keep taking the products which are often priced above $100,000 per treatment, then you will experience a reversal in your condition.

4. Gene therapy may not be able to adapt to a changing world.It has taken less than a century for prescription-grade antibiotics to no longer be as effective for the treatment of bacterial infections as it once was. Antibiotic resistance can impact anyone at any age, and in any country. Sometimes it occurs naturally, but the most common reason for this issue is that antibiotic misuse has led to a growing number of infections, including pneumonia, tuberculosis, and salmonellosis being more challenging to treat because the medicine is not as effective against the bacteria.

This issue could occur with gene therapies too. We have already seen people begin to have their progress reverse itself when they stop following their treatment plan. Over the next couple of generations, the body could start resisting this option too.

5. Gene therapy might only delay the inevitable.Jolee Mohr was lying in a Chicago hospital, her body swollen by internal bleeding and organ failure. The sight was so difficult that her husband decided not to bring their 5-year-old daughter into the room to say goodbye. Although there was no evidence to suggest a link, Jolee had taken an experimental treatment for rheumatoid arthritis. She was only 36 years old.

The National Institutes of Health approved the first human gene transfer study in 1989. Through 2006, there were 800 gene therapy studies that involved 5,000 patients. In those 17 years, the total number of approved therapies was zero. The only success story was a cure for the bubble boy disease that also caused leukemia thanks to the virus that delivered the treatment.

And Jolee wasnt the only story. A teenager named Jesse Gelsinger also died because of treatments offered inn a clinical research study. We must remember that there are sad stories to tell in addition to the happy ones when evaluating this treatment option.

6. Gene therapy will shift society toward new polarization.The United States is already highly polarized from a political perspective If gene therapies are approved for widespread use, then it may create another layer of separation from a medical perspective. Although most people can get behind the idea of creating a cure for cancer, birth defects, or chromosomal disorders, the processes used could also create designer genetics that promise a specific outcome. Should we pursue a scientific field that could help our children become smarter, faster, or better looking?

7. Gene therapy could change the way we think about competition.Although the discussion of designer babies often involves looks, the science behind gene therapy could also encourage specific traits to develop in children. Parents with wealth could work with their doctors to support a healthier muscle mass, faster fat burning capabilities, or an adaptive body frame that allows for greater flexibility in sports. People could design an outcome where results could follow a curve where outcomes could be planned for years in advance. This process would result in another layer of socioeconomic separation that would likely lead to even more polarization.

The pros and cons of gene therapy still require a lot of soul searching, even though we are 30 years and counting into this field of research. We are beginning to see some successes, but it has also come at the expense of some high-profile failures. Only time will tell if we can put this information to good use for the betterment of humanity. Until then, we must continue searching to find more solutions to the significant health issues our race faces each day.

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14 Advantages and Disadvantages of Gene Therapy - ConnectUS