Archive for the ‘Gene Therapy Research’ Category

Dublin, Jan. 06, 2021 (GLOBE NEWSWIRE) -- The "Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global cell & gene therapy market.

Major players in the cell and gene therapy market are Gilead Sciences, Bristol-Myers Squibb, Novartis AG, Amgen, Merck, Organogenesis Holdings, Dendreon, Vericel, Bluebird Bio and Fibrocell Science.

The global cell and gene therapy market is expected to decline from $6.68 billion in 2019 to $6.92 billion in 2020 at a compound annual growth rate (CAGR) of 3.61%. The slow growth is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The entire supply chain has been disrupted, impacting the market negatively. The market is then expected to recover and reach $13.23 billion in 2023 at a CAGR of 24.10%.

The cell and gene therapy market consists of sales of cell and gene therapies by entities (organizations, sole traders and partnerships) that develop cell and gene therapies. Cell therapy refers to the transfer of intact, live cells that are originated from autologous or allogenic sources and gene therapy refers to the introduction, removal, or change in the genome for treating diseases. The market consists of revenue generated by the companies developing cell and gene therapy products by the sales of these products.

North America was the largest region in the cell and gene therapy market in 2019. It is also expected to be the fastest-growing region in the forecast period.

In December 2019, Roche, a Swiss multinational healthcare company, acquired Spark Therapeutics for $4.3 billion. The acquisition supports the commitment of Roche to bring transformational therapies and innovative approaches to people with serious illnesses. Spark Therapeutics will continue to work within the Roche Group as an independent company. Spark Therapeutics, headquartered in Philadelphia, is a fully integrated commercial company involved in the discovery, production, and distribution of gene therapies for genetic disorders including blindness, hemophilia, lysosomal storage, and neurodegenerative diseases.

The cell and gene therapy market covered in this report is segmented by product into cell therapy; gene therapy and by application into oncology; dermatology; musculoskeletal; others.

Limited reimbursements preventing patients from receiving treatments are expected to limit the growth of cell and gene therapy (CGT market. In 2019, Trinity Life Sciences, a life sciences solution provider, researched national and large regional commercial health insurance plans in the US. It found that the confluence of increasing price, patient volume and number of CGTs on the market is likely to change the reimbursement model for CGTs and impact payer budgets by 5-10%. Payers realize that financing needs to be generated for cost management due to the uncertainty surrounding reimbursement of ancillary costs. Limited reimbursements and uncertain insurance plans are preventing patients from receiving high-cost CGT, which is expected to limit market growth.

Chimeric antigen receptor (CAR) T-cell therapy is shaping the cell and gene therapy (CGT) market. (CAR) T-cell therapy is a combination of cell and gene therapy in which T cells are collected from the patient's blood and are genetically engineered to produce modified receptors at their surface, known as chimeric antigen receptors (CARs). These modified T cells with special structures (receptors) are reinfused into the patient. Then, the modified receptors of T cell help in targeting the surface antigen of the cancer cell that ultimately results in the killing of tumor cells in patients.

In 2020, the US-FDA approved Bristol-Myers Squibb's two CAR-T cell therapies to treat lymphoma and multiple myeloma and is set to be launched. Currently, FDA approved CAR-T cell therapy treatments like Tisagenlecleucel for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL) in children and Axicabtagene ciloleucel for the treatment of adult patients with relapsed or refractory large B-cell lymphoma.

Steady investment and consolidation in cell and gene therapies contributed to the growth of the cell and gene therapy (CGT) market. After recognizing the potential of the CGT market, 16 out of the 20 largest biopharma companies by revenue, added CGT products to their portfolio.

Key Topics Covered:

1. Executive Summary

2. Cell And Gene Therapy Market Characteristics

3. Cell And Gene Therapy Market Size And Growth 3.1. Global Cell And Gene Therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Cell And Gene Therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Cell And Gene Therapy Market Segmentation 4.1. Global Cell And Gene Therapy Market, Segmentation By Product, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Cell And Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Cell And Gene Therapy Market Regional And Country Analysis

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/yvp1rq

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Link:
Global Cell and Gene Therapy Market Report 2020-2030: COVID-19 Impacts, Growth and Changes - GlobeNewswire

The hunt for effective therapies for solid tumors has heated up in early 2021 with a string of biotechs touting big investor checks and name-brand collaborations to chase those hard-to-treat lumps. Now, with one of its candidates already in the clinic for leukemia, Mana Therapeutics is taking a new round of funding to join the fray.

On Friday, Mana unveiled a $35 million Series A financing round that will help push the Boston-area biotechs lead candidate through a Phase I trial and could help the company secure an IND for an off-the-shelf allogeneic molecule targeting transplant-ineligible AML and solid tumors within the year.

The biotechs leading molecule, dubbed MANA-312, is already engaged in the Phase I study of patients with acute myeloid leukemia, myelodysplastic syndrome after undergoing an allogenic hematopoietic stem cell transplantation. Manas goal is to use its technology to create an inventory of off-the-shelf allogeneic products that can treat the majority of patients with certain targeted cancer indications using whats called a human leukocyte antigen matching system.

Its a different take on a similar line of attack for solid tumors: using the bodys natural immune system to educate healthy cells already in the body to target antigens on the surface of the tumors cancer cells without damaging the otherwise healthy cells. To do this, Mana uses an in-house platform called EDIFY, which it says leverages natural immune system pathways to educate T cells to target multiple cell surfaces and intracellular tumor-associated antigens.

Through multiple antigen targeting processes, the companys technology is designed to prevent the tumors immune escape, and it says the allogeneic method which uses healthy donor cells to create a master cell bank and is then used for specific therapies of attacking the solid cancer tumors could provide superior efficacy to single antigen and other cell therapy approaches.

MANA-312 also isnt the biotechs only candidate in the works. MANA-412 is a preclinical off-the-shelf allogeneic cell therapy for treating transplant-ineligible acute myeloid leukemia and solid tumors and could be ready for an IND filing by the end of the year, Mana said. The Series A round will help fund preclinical development for that candidate as well.

Mana was founded based on research and human proof-of-concept clinical trials conducted by Catherine Bollard of Childrens National Hospital and her colleagues at Johns Hopkins Medical Center. Those trials, in both solid and hematologic tumors, supported a strong safety profile, showed immunological anti-tumor activity and validated MANAs initial set of tumor antigens, the company said. Then Bollard co-founded the company with industry vet Marc Cohen. Ex-Gilead exec Martin Silverstein is the CEO.

The human proof-of-concept trials conducted by my team and colleagues showed potential for a nonengineered approach to educating T-cells to attack multiple tumor antigens, which MANA is expanding even further through refinement of the manufacturing process for an allogeneic product and application to a broader set of antigens in a variety of clinical indications and settings, Bollard said in a statement.

MANAs $35 million financing round was led by Cobro Ventures and Lightchain Capital and joined by LifeSci Venture Partners with other undisclosed investors.

See the original post:
Mana joins the hectic fight against solid tumors with an 'off-the-shelf' candidate angling for an IND this year - Endpoints News

Dive Brief:

As the name suggests, biomolecular condensates are tiny concentrations of molecules found inside cells. Scientists have observed these clusters, which take the form of liquid-like droplets, for decades, but only recently determined that they help regulate cellular reactions and activities.

Like other cell-managing structures, researchers suspect that biomolecular condensates can give rise to a variety of diseases if they malfunction. This thinking has led to the formation of several new drug companies in the last couple years, with Dewpoint being the first to come out of stealth mode.

Dewpoint arrived in early 2019, backed by the venture capital firm Polaris Partners and a string of other investors. Company leadership said the initial focus would be cancer and neurodegenerative diseases, though other areas like immunology and infectious disease also appear to be on Dewpoint's radar. The Boston-based biotech quickly drew interest from pharmaceutical giants, too, with Bayer and Merck & Co. inking separate deals potentially worth $100 million or more.

With the Pfizer deal, Dewpoint joins a handful of companies targeting DM1. Audentes Therapeutics, a gene therapy developer now owned by Astellas, has been exploring two approaches to treat the disease. Vertex and CRISPR Therapeutics also recently expanded a gene editing partnership to include DM1 and Duchenne muscular dystrophy.

Faze Medicines has its sights set on DM1 as well. The Cambridge, Massachusetts-based biotech debuted in December with $81 million in Series A funding, which was supplied, in part, by some big pharmaceutical firms, including the Novartis Venture Fund and AbbVie Ventures.

Faze is also trying to find and develop treatments for ALS. Interim CEO Cary Pfeffer recently told BioPharma Dive that Faze may take aim at other neurological and neurodegenerative disorders, as well as cancer, immune diseases and viral illnesses.

See the original post:
Dewpoint forges another big pharma partnership and a potential rivalry - BioPharma Dive

A little less than two years after bagging an extended $50 million Series C, IsoPlexis and its proteomic barcodes for personalized cancer care are back setting hooks to bring even more investors on board. And this time, theyve caught a big fish.

Perceptive Advisors is leading a $135 million Series D round for the company, the firms announced Thursday, comprising $85 million in equity and a $50 million line of credit. IsoPlexis plans to use the proceeds to expand commercial and R&D staff, increase operational capacity and accelerate product development.

The Branford, CT-based company works with cancer centers and biopharma companies in the US, Europe and China, using its biomarker-driven system designed to predict responses to such treatments and personalize treatment for patients. IsoPlexis employs a proprietary single-cell analysis tool to refine its immunotherapies.

We believe the future of advanced medicines will rely upon deeper access to in vivo biology for the development of new therapies and are excited to back the team at IsoPlexis, Perceptive portfolio manager Sam Chawla said in a statement.

Researchers have developed what they call proteomic barcode chips, which allow them to look at the entire complement of proteins within a patients cells. Its a process they say provides the mapping of new and accessible layers of biological data for every single cell, ultimately allowing for a better understanding of how individuals may respond to therapies.

Essentially, patients receive samples of these chips, which CEO Sean Mackay says is barcoded with antibodies. After the company receives the sample back, they place it into their system to see just how a persons immune system would respond to different treatments.

We call that the single-cell immune landscaping, Mackay told Endpoints News. What were able to do with that is find subsets of powerful immune cells that you typically miss in bulk profiling, sort of status quo, and that is a product that works on our instrument, basically a software-enabled system that reads out what the chips look like and what the proteins are per cell.

That software then lets IsoPlexis compare whats typically missed in that bulk profiling to long-term responder patients in several different fields like cancer immunotherapy, cell and gene therapy, Covid-19 and autoimmune disease, among other areas. IsoPlexis can then pick and choose the appropriate preclinical treatments and biomarkers in the clinic, packaging that info to pharma companies and academic labs.

Perceptive, historically a passive investor that enjoys clinical-stage investments and crossover rounds, has been fairly busy over the last year or so. It made its first foray into the company formation and Series A spaces in late 2019, setting up a $210 million early-stage VC fund with Xontogeny. Then last August, they launched their first in-house start-up in China, followed by a $310 million raise a few months later. Perceptives third SPAC also filed for an IPO in late July.

Other new investors included Ally Bridge Group and funds and accounts managed by BlackRock. Unnamed existing investors also participated in the round.

Here is the original post:
IsoPlexis scores big backer for personalized protein 'barcodes' as Perceptive jumps on board new funding round - Endpoints News

LONDON--(BUSINESS WIRE)--The soft tissue repair market is poised to grow by USD 10.44 billion during 2020-2024, progressing at a CAGR of over 11% during the forecast period.

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The report on the soft tissue repair market provides a holistic update, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis.

The report offers an up-to-date analysis regarding the current global market scenario and the overall market environment. The market is driven by the rising incidence of accidental injuries.

The soft tissue repair market analysis includes Product segment and Geography Landscape. This study identifies the growing demand for gene therapy as one of the prime reasons driving the soft tissue repair market growth during the next few years.

This report presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters.

The soft tissue repair market covers the following areas:

Soft Tissue Repair Market SizingSoft Tissue Repair Market ForecastSoft Tissue Repair Market Analysis

Companies Mentioned

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Medical Textiles Market by Application, Product, and Geography - Forecast and Analysis 2020-2024: The medical textiles market size will grow by USD 2.99 billion during 2020-2024. The markets growth momentum will accelerate during the forecast period because of the market drivers, which have been explored in this report. To get extensive research insights: Click and Get FREE Sample Report in Minutes!

Key Topics Covered:

Executive Summary

Market Landscape

Market Sizing

Five Forces Analysis

Market Segmentation by Product

Market Segmentation by Application

Market Segmentation by End-user

Customer landscape

Geographic Landscape

Vendor Landscape

Vendor Analysis

Appendix

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Global Soft Tissue Repair Market- Featuring 3M Co., Arthrex Inc., and Baxter International Inc. Among Others - Business Wire

In an effort to crack the code of hard-to-treat solid tumors, biopharma has tried numerous pathways to effectively target those masses without damaging healthy tissues. Phillys Carisma Therapeutics thinks it has a winner with its macrophage cell-based CAR-M candidates, and now its taking a new flush of investor cash to try one in the clinic.

Carisma has scored a $47 million Series B round to take its lead candidate, anti-HER2 CAR-M tumor fighter CT-0508, into a Phase I trial as well as advancing the rest of its preclinical macrophage pipeline. Carisma has keyed in on the use of targeted macrophage cells and vectors to penetrate the environment of solid cancer tumors without hurting health tissues a puzzle in the solid tumor field.

CT-0508s early-stage study will turn out the first human data for a CAR-M therapy based on those macrophage cells, Carisma said. CEO Steven Kelly told Endpoints News his company could offer an effective and safer way to target tumors and warm them up for the immune system to attack.

There are a number of characteristics about macrophages that would lend themselves towards applications of solid tumors and other indications, but what were focused on is solid tumors, Kelly said. Macrophages are preferentially recruited into a tumor microenvironment, and lymphocytes like the CAR-T approaches are actively excluded. So we think that we have an advantage by overcoming trafficking limitations to solid tumors.

This has been the sticking point for the industry: therapeutics that can invade the walls which surround cancerous tumors without damaging otherwise healthy cells.

Kelly is confident that Carismas technology will ultimately decipher how to do just that.

Once it starts eating (the cancerous tumor cells), the macrophages will start producing cytokines that effectively warm up that environment and convert an immunologically cold tumor into a warm or hot tumor and recruit in other cells, like T cells for example. So that last element is really unique to macrophages due to the antigen presentation capability, he said. They engage in cells directly, they warm up the tumor microenvironment, and they generate a true adaptive immune response. Thats how we think of ourselves and how were differentiated in the cell therapy space.

Kelly said it was a bit premature to know when Carisma would begin public readouts of the data surrounding its macrophage therapeutics, but he hoped they would be able to do so by the middle of this year.

The total capital Carisma has raised since its Series A financing in 2018 now totals roughly $109 million, and is a key step in moving the company from effectively a discovery-stage company to a clinical-stage company, Kelly said.

A lot of effort has gone into building this company. We had to transition from a bench project at (the University of) Penn, we had to demonstrate all the things necessary to get an IND declared (so) safety and efficacy we had to develop a GMP manufacturing process, Kelly said. All those were effectively developed to FDA satisfaction, and were moving into the clinic now.

Investors in the Series B financing are led by Symbiosis II, with subsequent investment from Solasta Ventures and Livzon Pharmaceutical Group. Additionally, Carisma said, existing investors AbbVie Ventures, HealthCap, Wellington Partners, IP Group, TPG Biotech, Agent Capital, and MRL Ventures Fund contributed to the new round of funding.

Read more from the original source:
Looking to solve the solid tumor puzzle box, Carisma aims to take 'CAR-M' groundbreaker into early-stage trial - Endpoints News

Investors like to see big plans, and Kevin Judice has plenty. The DiCE Molecules CEO is plotting a clinical trial launch for the biotechs lead small molecule for psoriasis and wants to double the staff in the next year and a half.

On Friday, those big plans landed him an $80 million Series C round.

Were very excited about it, he said of the raise led by RA Capital Management.

The round comes around two years after a $50 million Series B. While the B round was used for optimizing technology and building a pipeline, Judice says the Series C will propel the biotechs IL-17 antagonist to the clinic and fund the development of two other undisclosed programs.

This new capital allows us to expand our reach and get at more targets and have more opportunities to make high impact, Judice said.

DiCEs development process revolves around its DNA-encoded library. Such libraries allow researchers to screen millions even billions of compounds in parallel, using DNA tags that Judice compared to barcodes, which tell you what the constituent pieces of a molecule are.

Usually you do some kind of screen, like a high-throughput screen, or a DNA-encoded library screen, something like that, and you get a few hits. And then theres a long phase of just lab chemistry, where youre making individual compounds and trying to progress those hits, those initial binders, to something thats closer to a drug, Judice said.

That hit-to-lead phase is typically labor-intensive and slow, the CEO said. But DiCEs approach accelerates that work by using a smaller DNA-encoded library much smaller but richer in information, Judice said to screen in different ways after getting a hit.

What were actually looking for is the difference between just binding and something that is functional, Judice said.

DiCEs lead program is an agonist for cytokine receptor IL-17, which is implicated in diseases like psoriasis and psoriatic arthritis. Current antibody treatments targeting IL-17 are quite effective at treating psoriasis, but they are injectable and lack in convenience. DiCEs candidate would be oral, and the biotech is hoping to top the efficacy of Amgens already approved oral PDE4 inhibitor Otezla.

What were working on is an oral that will work as well as the anti-IL-17 antibodies. So it combines the convenience and safety of something like Otezla with the efficacy of an antibody like Cosentyx, Judice said. The antibodies tell us that IL-17 is exactly the right target.

Since 2017, DiCE has grown from a seven-person, peanut-sized company to a 29-person staff. And in the next 18 months, Judice is looking to bring that number to 58. The biotech inked a $2.3 billion discovery pact with Sanofi years ago, and is currently partnering with them on an I-O small-molecule program that Judice says isnt far behind the IL-17 candidate.

We should be ready to go public with more data on earlier programs over the course of the next 12 months. And then Im really excited about the opportunity to grow the pipeline by adding new programs to it, he said. Thats one of the things that is particularly great, from my perspective, about having RA Capital lead this round.

In addition to RA, Eventide Asset Management, New Leaf Venture Partners, Soleus Capital, Driehaus Capital Management, Osage University Partners and Asymmetry Capital Management, Northpond Ventures, Sands Capital, Sanofi Ventures, Alexandria Venture Investments, Altitude Ventures and Agent Capital also chipped into the Series C.

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DiCE gets its 'library' card ready as it speeds development of DNA database-derived molecules with more investor cash - Endpoints News

Dec. 28 (UPI) -- Fewer than 2% of people with treatment-resistant high blood pressure are checked for a hormone disorder that can drive blood pressure higher, a study published Monday by the Annals of Internal Medicine found.

In people with primary aldosteronism, the adrenal gland produces too much of a hormone called aldosterone, causing increases in blood pressure, often to unhealthy levels, according to the Mayo Clinic.

Professional guidelines recommend checking those with high blood pressure that doesn't respond to treatment for the hormonal disorder, but researchers found the tests often are not performed.

"While primary aldosteronism is a common cause of difficult to control hypertension, it is under-diagnosed," study co-author Dr. Jordana Cohen told UPI.

"Patients who were tested in our study were more likely to be treated with the appropriate medications ... and to have better blood pressure control over time, said Cohen, an assistant professor of medicine and epidemiology at the University of Pennsylvania.

Many people who don't respond well to commonly used blood pressure control medications, including beta blockers and ACE inhibitors, are found to have the hormone disorder, Cohen and her colleagues said.

The condition has been linked with a four- to 12-fold increased risk for cardiovascular events, such as heart attack and stroke, compared to those with high blood pressure due to other causes, the researchers said.

However, the disorder can be effectively treated with drugs called mineralocorticoid receptor antagonists, or MRAs, including spironolactone and eplerenone, or surgery, according to Cohen.

For this study, she and researchers at the University of Pennsylvania, Stanford University and the University of Michigan reviewed data from the Veterans Health Administration for more than 269,000 veterans with apparent treatment-resistant high blood pressure.

Treatment-resistant hypertension was defined as either two blood pressures of at least 140 systolic or 90 diastolic at least one month apart. Patients also had to be receiving treatment with at least three blood pressure drugs, including a diuretic, or with at least four different types of blood pressure drugs, the researchers said.

Just under 2% of patients with treatment-resistant high blood pressure underwent guideline-recommended testing for the hormone disorder, the data showed.

Testing rates ranged from 0% to 6% at centers included in the study and did not correlate with the number of patients with treatment-resistant hypertension, the researchers said.

And just 15% of the patients were on an MRA drug, the data showed.

Patients tested for the hormone condition were more likely to receive treatment with MRAs and have better long-term blood pressure control, according to the researchers.

Testing rates also did not change meaningfully over nearly two decades of follow-up despite an increasing number of guidelines recommending testing for the disorder in this population, they said.

"If you are on three or more medications for management of your blood pressure, ask your doctor if they think you might benefit from testing or from being treated with an MRA," Cohen said.

"Not all patients are appropriate to be tested or treated with MRAs, but most people with treatment resistant hypertension are," she said.

Read the original:
Study: Too few with high blood pressure tested for hormone disorder - UPI News

Over the last several years, parents from across the country have appeared on television and news outlets to raise money to develop treatments for their children with rare genetic disorders.

Many of these families, including one from Kansas City, Missouri, have pinned their hopes on one Texas researcher, Dr. Steven Gray of the University of Texas Southwestern Medical Center in Dallas, who claims to be on the verge of treating a number of rare conditions. They've raised millions of dollars to fund his research, although breakthroughs haven't happened to the extent many had hoped.

While gene therapy holds great promise, the growing trend of family-funded research concerns some medical ethicists, who say that suggestions to parents that treatments may be imminent can raise thorny ethical issues.

These are parents. They are desperate. says Mayo Clinic bioethicist Megan Allyse. They are willing to try almost anything. They are in a pretty vulnerable position for somebody to come along and say, Give me your money, and I can make this better.

Further complicating the picture is the recent entry into the field of a private company, Taysha Gene Therapies, which says it will accelerate Grays research. That has divided his supporters and raised concerns about families who might be left behind.

Kim Fry, of Kansas City, Missouri, has a video on her phone of her son, Charlie, that was made in 2018 when he was 5 months old. It shows her bright-eyed boy gently shaking in his father's lap, as if shivering from cold.

Those little tremors sent the family on a year-long odyssey that led to a frightening diagnosis. A genetic test showed Charlie had an incredibly rare mutation in a single gene, SLC6A1. The mutation typically causes intellectual disabilities and epilepsy starting around the age of three-and-a-half that can severely affect patients for the rest of their lives.

Doctors told Kim and Nate there was no treatment available to help their son.

At that moment, you just feel crushed and kind of begin grieving for the life you think your childs going to have, Kim said.

Rare genetic disorders have generally received little attention from biotech companies because the markets for treatments are so small.

But shortly after the diagnosis, Kim met Amber Freed, a mother from Denver who seemed to have found a solution. After her son, Maxwell, was diagnosed with the same mutation a year earlier, Freed met Gray, a molecular biologist who had focused on gene therapy while doing a post-doctoral fellowship at UNC Chapel Hill.

Gray was developing treatments or even possible cures for conditions caused by single-gene mutations, and he had agreed to work on SLC6A1. But it would be up to Freed to provide Gray with the millions of dollars he would need to do this work.

With the possibility of a treatment suddenly on the table, Kim and Nate immediately joined Amber in raising money through the organization she started, SLC6A1 Connect, and their own campaign, A Cure For Charlie. Their goal was to create a treatment and bring it to clinical trial before Charlie turned three-and-half, hoping to block the severe effects of the mutation and giving him a chance to live a regular life.

Once the funding is there, then all the science is going to move into place, so really the only hurdle that were facing right now between us and the cure is the funding, Nate said in fall of 2019.

Family fundraisers

Family fundraisers are a departure from how medical research is usually funded, typically through the National Institutes of Health, large foundations and advocacy groups.

Nevertheless, families like the Frys have appeared on news outlets and in publications throughout the country in recent years, from Good Morning America and ABC News to People magazine and countless local news shows, to raise money for research.

The children have been diagnosed with many different genetic disorders, but their stories are similar. They all have rare gene mutations that will lead to serious mental or physical declines or early death, and their parents are pinning their hopes on Gray to develop treatments.

They call themselves the Steve Gray Parents.

Gray himself has appeared in articles and videos, including one produced by UT Southwestern about Willow Canaan, a girl from Mississippi who has multiple sulfatase deficiency.

A lot of the families that we interact with, they are coming to us with really sick kids. I think knowing their story, knowing that one child, gives us a face, gives us a mission that if we can move fast enough theres hope that we could treat and we make things different for that specific child, Gray said in one video.

Gray became a go-to researcher for rare disease families after his treatment of Hannah Sames, a girl from New York with a degenerative genetic disorder called giant axonal neuropathy.

In a 2016 clinical trial, Sames was injected with a manufactured virus that contained a working copy of the gene that was mutated. Through this adeno-associated virus delivery method, the normal gene would take over from the mutated one and stop the degeneration from happening.

The treatment slowed the progress of Hannahs disease, according to Gray, but it wasnt a cure. In a 2019 interview on Connecticut Public Radio, Hannahs mother, Lori, said the family was seeking additional treatment.

But Gray thinks the same method could be used to treat and possibly cure all kinds of genetic disorders, including the SLC6A1 mutation

Were due for another leap in technology, Gray told KCUR in 2019. Were going to have a better virus technology, better ways to deliver genes, and I can see that just making a further leap for the whole field.

Gray, who says he has been involved in developing treatments for two dozen diseases, has accepted money from families to pay for the high costs of manufacturing viruses, doing toxicology studies and running clinical trials. Many of these family groups had raised more than a $1 million each from their friends, relatives and neighbors.

Though these families have been effective at raising money, bioethicist Allyse worries that without the peer review process that traditional funders use, they may not be in the best position to decide what research is likely to get results.

The potential problem with going around that process is that its possible to sort of go down avenues that are less supported by the literature, that are less in line with the scientific consensus, Allyse said.

But those doubts have done little to discourage dedicated parents like Amber Freed.

In early December 2019, Freed hosted the second annual SLC6A1 research symposium in a hotel conference room in downtown Baltimore. Freed quit her job in finance after her sons diagnosis to dedicate herself to advancing a treatment, and she began organizing annual SLC6A1 research symposiums in 2018 to drum up interest in the work.

Shes also held charity golf tournaments, set up fundraising campaigns with companies like Amazon and Pizza Hut, and helped arrange the creation of genetically altered mice in China for research.

During the last two years, Freed has cultivated relationships with genetic researchers from all over the world, and as the sleepy scientists who traveled to Baltimore to take part in the symposium wandered into the conference room early on a Friday morning, she greeted them like family, with big hugs and smiles.

Alex Smith

Despite Freeds seemingly endless enthusiasm, she made clear in her welcoming speech to the scientists that, unlike them, her involvement in SLC6A1 research didnt happen by choice.

But to be honestI dont want to be here, read a slide projected behind her at the end of her remarks.

Some improvements but no 'home run'

Toward the end of the day, Gray took the podium to deliver an update on research from his lab. For many in the audience, this was the days main event.

His teams early research using the treatment showed some improvements in motor and behavioral skills in young, genetically altered mice that were treated before symptoms had appeared. But there was no change in mice that already had symptoms.

I think treating at an early age, were seeing some signs of improvements and some nice signals that our vector is doing something positive, but, you know, its not a home run, Gray explained.

Though it wasnt the result Freed dreamed of, she was encouraged that the research appeared to be on the right path.

Gray insisted he had tried to be careful about managing expectations for families funding his work, but between symposium talks, he also said he had recently shifted course on working with them.

Im really having to say no a lot now, Gray told KCUR. Im kind of moving into a point where we were trying not to say no, and we were trying to work on everything that the science made sense. But there is a point where you just have to say, You know Ive got to focus on what Im doing, and theres a limit.

While Grays work did lead to a treatment for Hannah Sames, similar breakthroughs havent come in time for other families.

Laura King Edwards of Charlotte, North Carolina, started working with her family to raise money for Grays work after her baby sister, Taylor, was diagnosed with a form of a rare disorder called infantile Batten disease in 2006.

The family didnt have a lot of hope the research would lead to a treatment in time to help Taylor, and she died two years ago at age 20.

Edwards says that looking back, she sometimes regrets all the time she spent running a fundraising organization.

Id spend hours up at night dealing with tech issues on our website, for example, or responding to emails from people all over the world, knowing that thats time that maybe I couldve spent with my little sister while she was still here, Edwards says.

Nevertheless, even after Taylor was gone, her family continued to support Grays work through their organization, Taylor's Tale.

A new player

Not long after the conference in Baltimore, however, the race for a SLC6A1 treatment slowed to a crawl.

When the COVID-19 pandemic hit, scientific studies and medical trials across the country were stopped and research funds were directed to coronavirus research.

The therapists who work with Amber and Kims sons were unable to meet with them in person, and the boys started to backslide on some of their developmental progress.

Then, after the initial waves of the coronavirus subsided, hopes for Grays research came roaring back to life when a new company, Taysha Gene Therapies, announced it would partner with UT Southwestern, offering a boost to the research and development beyond what families could provide.

They could get it to a certain place, said Taysha founder R.A. Session II, But when it needs to get to kind of the meaningful level in order to get it into late-stage clinical trials, this is where they just dont necessarily have the capability. And so I think this is where you would see programs then transitioning into a companys hands in order to kind of pursue them and move them forward.

In April, Taysha announced a partnership with UT Southwestern that would fund Grays research and work to move it more quickly into clinical trials and possible treatment. Gray was named chief scientific officer.

The company said the family fundraising would no longer be needed.

For some parents, like Doug and Kasey Woleben of Dallas, that was great news. Theyve raised around $1 million for research to treat Leigh syndrome, which affects their 8-year-old son, Will.

We were excited, thrilled to know that were now off the hook for millions and millions and millions of dollars. And that Taysha and UT Southwestern are trying to push this program and move it forward as quickly as possible. So for us, it was a miracle, Kasey said.

But Taysha's involvement and its timeline have brought disappointment for other families. The company's first clinical trials, to treat a mutation that causes Tay Sachs disease, were planned to start in Canada at the end of 2020 but only received approval from the Canadian government this week.

The company says it plans to seek permission to test treatments for three other conditions, including the SLC6A1 mutation, by the end of 2021, but it has not announced any dates for beginning trials.

For Amber Freed and Kim Fry, Tayshas timeline is problematic. Both of their sons were expected to exhibit epilepsy symptoms before the end of next year and so they would see little benefit from treatment initiated after that.

Im very disappointed, Freed said in September. If you had asked me this time last year, I would have fully expected to be in a clinical trial right now.

Different priorities

Session insists that Tayshas timeline and priorities on are based on what the research shows is safe and effective.

Weve allowed the science to kind of move forward at the pace the science moves, Session says. Then we move it forward into the clinic based on that science.

But to Freed, the goal of fast-tracking to trial, even one that would have only resulted in a slight improvement for her son, appears much less likely now that Taysha is involved.

Once you hand over the reins to a biotech, you lose decision-making power as a nonprofit organization, and you abide by their timeline and not necessarily your own, Freed said. In my case, we are racing to get this therapy into children like Maxwell and Charlie as quickly as possible, so we need it done tomorrow.

For other Gray supporters, however, the future is even less clear. Tayshas development pipeline does not include treatments for some of the conditions that Gray had previously been working with families to develop, including Charcot-Marie Tooth, Krabbe disease and multiple sulfatase deficiency.

UT Southwestern researchers will continue to research those conditions, according to a university spokesman, and Taysha says it plans to expand its pipeline in the future.

Terry Pirovolakis, who had enlisted Grays help to develop a treatment for spastic paraplegia 50, which affects his son, Michael, will not be involved with Tayshas work. Hell only continue to work with UT Southwestern directly.

From my perspective, it was, thats great. Tayshas gonna come in and maybe save the world, but I dont want to be part of it 'cause theyve got a lot of stuff they gotta work out, and Im not going to wait around for them to figure it out, Pirovolakis said.

Pirovolakis, who lives in Toronto, has raised more than $1.5 million since May 2019 through his online campaign, Cure Michael, which was the most successful GoFundMe campaign in Canada last year.

Expectations vs. reality

He says that while he has been comfortable working with Gray, he believes that drug companies, which depend on the involvement of families for rare disease research, can mislead parents about what might be possible for their children.

The industry, as a whole, I think, maybe sets expectations that are higher than reality," Pirovolakis said. "We see these presentations at the conferences of these kids doing amazing things, like a 4-year-old that has no brain function pretty much, going to school two years later. Its remarkable.

"But that was five or 10 years of research. So I think that expectations from the industry are maybe what cloud us as parents in the hope that something amazing is gonna happen for our kids.

CureCMT4J, a foundation created to advance research on Charcot-Marie Tooth by parent advocate Jocelyn Duff, an early supporter of Gray, also is no longer involved with the researchers work. Duff said the organization had moved in a different direction, but she declined the provide details. The group had raised $1.3 million as of fall 2020.

Some ethicists have also raised questions about the costs of rare disease treatment, and they point to a drug previously developed by members of the Taysha team as a prime example of their concern.

Several members of the Taysha team, including Session, directors Sean Nolan and Phillip Donenberg, and others, earned their reputation for success as part of a AveXis, a company that developed the breakthrough treatment for spinal muscular atrophy, Zolgensma.

Zolgensma was introduced by drug giant Novartis last year with a price tag of more than $2.1 million, making it the most expensive drug in the country.

On the one hand, you could say thats a winning team, said Megan Allyse. On the other hand, you could say is that the team you want to be on if what youre trying to do is generate not just effective treatments, but accessible treatments?

Session says that Taysha currently has no plans regarding the pricing or accessibility of any treatments the company might develop.

We should be so blessed to be able to have a discussion on pricing because then were talking about an approved therapy, Session says. But were not there yet. So what I would say is the company is focused on getting these drugs into patients effectively and safely as efficiently as possible.

Taysha announced in November that it raised more than $275 million in private financing and an initial public offering.

For Kim Fry and Amber Freed, however, the focus is still very much on what can be done for their sons.

The women are continuing to raise money, but they have shifted to other researchers and technologies. And they have adjusted their expectations.

Frys son, Charlie, started having more significant seizures earlier in the year and is now taking medication to reduce them.

She still thinks a treatment within the next year or two could help her son and others like him, although not in the way she had once imagined.

It may not be a 100% home run where they live 100% the life we hoped. But theyll still have a better life than they are living today, Fry said. I lose sleep every night over the thought that it might be too late, but Im still hopeful that there will be benefit for them.

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These Families Raised Millions To Fund Treatment For Their Kids' Genetic Disorders. It Hasn't Happened. - KCUR

TSHA-101 will be the first bicistronic vector to enter a first-in-human clinical study, which is a significant milestone for Taysha and for the field of gene therapy, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. GM2 is a devastating lysosomal storage disease with no approved treatments and todays CTA approval marks a formative moment for children suffering from this rapidly progressive and fatal disease.

The trial will be a single arm, open-label Phase 1/2 trial evaluating the use of TSHA-101 for the treatment of infants with GM2. The study will be sponsored by Queens University and led by Jagdeep S. Walia, MBBS, FRCPC, FCCMG, Clinical Geneticist and Associate Professor Head, Division of Medical Genetics (Department of Pediatrics) at Queens, and Director of Research (Department of Pediatrics), at the Kingston Health Sciences Centre.

Preclinical evidence to date supports our belief that TSHA-101, when given intrathecally as a bicistronic transgene packaged into a single AAV9 vector, has the potential to address the lysosomal enzyme deficiency, to change the disease trajectory and to improve patient survival, said Dr. Jagdeep S. Walia. We are pleased to have the support of Health Canada as we continue to advance TSHA-101.

Todays CTA approval is a culmination of our teams and Dr. Walias tireless efforts and a momentous occasion for children affected by GM2 along with their parents and caregivers, said RA Session II, Founder, President and CEO of Taysha. We are grateful to our partners at Queens University for their work to advance this gene therapy into the clinic.

About GM2 Gangliosidosis

GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the -hexosaminidase A enzyme. These genetic defects result in progressive dysfunction of the central nervous system. There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.

About TSHA-101

TSHA-101 is an investigational gene therapy administered intrathecally for the treatment of infantile GM2 gangliosidosis. The gene therapy is designed to deliver two genes HEXA and HEXB driven by a single promoter within the same AAV9 construct, also known as a bicistronic vector. This approach allows the simultaneous expression of a 1:1 ratio of the two subunits of protein required to generate a functional enzyme. It is the first and only bicistronic vector currently in clinical development and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA).

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-101, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Announces Queen's University's Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of...

The argument for continued investment

C> is a high potential and maturing sector, and is an already crowded environment, playing host to numerous start-ups and now, through M&A, recognised big pharma firms. Much like the rush to find a COVID-19 vaccine that dominates headlines worldwide, not every company involved will be able to succeed.

But finnCaps finnLife watch list of 50 leading AIM-listed biotech companies demonstrates that there is room for numerous companies to contribute to, and profit from, C>. Examining three entirely different approaches to CAR-T therapy, it is possible to see just how much space there is for this exciting sector, therefore displaying the case for continued investment.

Innovative CAR-T therapy demonstrates the depth of C> potential

CAR-T therapy in its existing form is a relatively new and specialised approach at treating cancer. It takes T cells from a patients bloodstream and genetically modifies them in a laboratory. These T cells are then injected back into the bloodstream with the aim of targeting and killing cancer cells.

While it has been shown to be an effective treatment, there are risks and side effects. One is the two-step autologous process (the slow time it takes for cell expansion sometimes as long as two weeks) while another is cytokine release syndrome (CRS), which occurs when cytokine molecules are inadvertently released, but too quickly to target just the tumours and instead target healthy cells.

The next generation of CAR-T treatments shows that there is space for a multitude of start-ups to be active in the C> space as they all help find varied solutions to these problems without negating the effectiveness of CAR-T.

One example is Horizon Delivery, a company that is developing its CYAD-02 project, which will help transport T cells more effectively to the tumour via the use of SMARTvector products.

The product underwent its first phase 1 trial test in January 2020 with a patient who was suffering from acute myeloid leukaemia. Horizon Delivery is also an industry leader in CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) screenings, meaning they can identify key genes or genetic sequences that draw out specific functions of a cell type from thousands of potential variants.

In a cancer context, this means they can route out and exclusively eliminate problematic cells that may have shown signs theyd resist a future cancer treatment.

Another example is Maxcyte, a global cell- based therapies and life sciences company that is developing its CARMA process, where a patients peripheral blood mononuclear cells (PBMCs) are removed and modified. The modified cells can then be used to target an array of different cancers.

Currently the company is conducting a phase 1 trial for advanced ovarian cancer in a dose escalation trial that will treat four separate cohorts the fourth of which was administered in March 2020.

Another example which shows the versatility of new CAR-T innovation is provided by Oxford Biomedica, a gene and cell therapy company specialising in the development of gene-based medicines.

Rather than a contained project or platform, its contribution to CAR-T is through a contract manufacturing development organisation. Collaborating with pharma companies, Oxford Biomedica uses its infrastructure to produce other companies licensed products, including Novartis Kymriah treatment (alongside other undisclosed CAR-T-related products).

With fast-moving innovation finally allowing multiple C> treatments to gain regulatory approval, along with a huge pipeline of upcoming therapies and an influx of funding and M&A activity, investing in C> no longer entails taking a bet on potential the future is finally here.

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After years of potential, cell and gene therapy is ready for the pharmaceutical mainstream - PMLiVE

Dublin, Dec. 23, 2020 (GLOBE NEWSWIRE) -- The "Regenerative Medicine in Pharma - Thematic Research" report has been added to ResearchAndMarkets.com's offering.

Regenerative medicine is a multidisciplinary field that seeks to develop the science and tools that can help repair, augment, replace, or regenerate damaged or diseased human cells, tissues, genes, organs, or metabolic processes, to restore normal function. It may involve the transplantation of stem cells, progenitor cells, or tissue, stimulation of the body's own repair mechanisms, or the use of cells as delivery vehicles for therapeutic agents such as genes and cytokines.

It is widely anticipated that Gene therapy is the most valuable regenerative medicine sector however, this market is also expected to be slowed down by high cost of therapies, which may limit its accessibility.Existing programs will facilitate the approval and development of regenerative medicines, however, a reimbursement system especially for curative therapies is warranted.

The Regenerative Medicine in Pharma report combines primary research from a cross-specialty panel of experts with in-house analyst expertise to provide an assessment of the development landscape.

This report assesses -

Scope

Key Topics Covered:

For more information about this report visit https://www.researchandmarkets.com/r/7trz9r

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Regenerative Medicine in Pharma 2020 - Opportunities, Challenges, and Unmet Needs - GlobeNewswire

Global Gene Therapy for Rare Disease Market Report, Sales and Consumption Status and Prospects Professional Research, the report classifies the Global Gene Therapy for Rare Disease Market in a precise manner to offer detailed insights about the aspects responsible for augmenting as well as restraining market growth.

Gene Therapy for Rare Disease Market report provides a thoroughly researched abstract of the key players with considerable shareholdings at a global level regarding demand, sales, and income by providing better products and services. Research Report outlines a forecast for the Gene Therapy for Rare Disease market between 2020 and 2027. In terms of value, the Gene Therapy for Rare Disease industry is expected to register a steady CAGR during the forecast period.

In recent past, most of the gene therapies received orphan drug designations. Orphan drugs are generally defined as those medicines with one or more indications approved under the Orphan Drug Act of 1983. The Orphan Drug Act supports the development of innovative treatments for rare disease patients. The creation of the orphan drug designation with the passage of the Orphan Drug Act in 1983 has facilitated the development and approval of drugs for rare diseases and 2017 and 2018, were marked by the highest number of orphan drug and indication approvals to date. Production of gene therapies is associated with use of high-end technologies, high research and development costs, and skilled scientists and researchers, which reflects in high prices of these therapies.

Note: *The Download PDF brochure only consists of Table of Content, Research Framework, and Research Methodology

Get PDF Brochure Of This Research Report @ https://www.coherentmarketinsights.com/insight/request-pdf/2321

The key players profiled in this report include: Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.

Regions included:

o North America (United States, Canada, and Mexico)

o Europe (Germany, France, UK, Russia, and Italy)

o Global (China, Japan, Korea, India, and Southeast Asia)

o South America (Brazil, Argentina, Colombia)

o The Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

Key Benefits:

o This study gives a detailed analysis of drivers and factors limiting the market expansion of Gene Therapy for Rare Disease

o The micro-level analysis is conducted based on its product types, end-user applications, and geographic

o Porters five forces model gives an in-depth analysis of buyers and suppliers, threats of new entrants & substitutes and competition amongst the key market players

o By understanding the value chain analysis, the stakeholders can get a clear and detailed picture of this Gene Therapy for Rare Disease market

Table of Contents

Report Overview: It includes the Gene Therapy for Rare Disease market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary: This section of the report gives information about Gene Therapy for Rare Disease market trends and shares, market size analysis by region and analysis of Global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players: Here, key players of the Gene Therapy for Rare Disease market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study: All of the regions and countries analyzed in the Gene Therapy for Rare Disease market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

The research study can answer the following Key questions:

What will be the progress rate of the Gene Therapy for Rare Disease Market for the conjecture period, 2020-2027?What are the prominent factors driving the Gene Therapy for Rare Disease Market across different regions?Who are the major vendors dominating the Gene Therapy for Rare Disease industry and what are their winning strategies?What will be the market scope for the estimated period?What are the major trends shaping the expansion of the industry in the coming years?What are the challenges faced by the Gene Therapy for Rare Disease Market?

Major Highlights of TOC:

Chapter One: Global Gene Therapy for Rare Disease Market Industry Overview

1.1Gene Therapy for Rare Disease Industry

1.1.1 Overview

1.1.2 Products of Major Companies

1.2Gene Therapy for Rare Disease Market Segment

1.2.1 Industry Chain

1.2.2 Consumer Distribution

1.3 Price & Cost Overview

Chapter Two: Global Gene Therapy for Rare Disease Market Demand

2.1 Segment Overview

2.1.1 APPLICATION 1

2.1.2 APPLICATION 2

2.1.3 Other

2.2 Global Gene Therapy for Rare Disease Market Size by Demand

2.3 Global Gene Therapy for Rare Disease Market Forecast by Demand

Chapter Three: Global Gene Therapy for Rare Disease Market by Type

3.1 By Type

3.1.1 TYPE 1

3.1.2 TYPE 2

3.2Gene Therapy for Rare Disease Market Size by Type

3.3Gene Therapy for Rare Disease Market Forecast by Type

Chapter Four: Major Region of Gene Therapy for Rare Disease Market

4.1 Global Gene Therapy for Rare Disease Sales

4.2 Global Gene Therapy for Rare Disease Revenue & market share

Chapter Five: Major Companies List

Chapter Six: Conclusion

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Gene Therapy for Rare Disease Market Prospects Pinpoint Higher Traction from Developed Nations during 2020-2026 | Coherent Market Insights | Kite...

Global Gene Therapy Market Research Report provides an complete recent proceeding in the market. The analysis supplies important data with figurative tables, graphs, charts, and statistics, an in-depth analysis of the market. The analysis highlights this markets fundamental dynamics for the forecast period (2020-2026), involving the trends, opportunities, restraints, and a lot more. The Gene Therapy analysis introduces a thorough evaluation to forecast the current market size, share, value, volume, gross sales, drivers, restraints, opinions by industry experts, and invaluable insights by the industrys prospective rise.

Every section of this report particularly consists of the research key elements of the market. The Gene Therapy industry dynamics segment permeates deep by the drivers, restraints, trends, and opportunities from this market. The quantitative and qualitative analysis, we help you with detailed and in-depth research on the Gene Therapy market. We also have centered on SWOT, PESTEL, along with different analysis of the industry.

Checkout FREE Report Sample of Gene Therapy Market Report for Better Recognizing: https://www.futuristicreports.com/request-sample/106731

(Kite Pharma Inc., Spark Therapeutics Inc., Novartis, GlaxoSmithKline PLC, Applied Genetic Technologies Corporation, NewLink Genetics Corp, Transgene SA, Oxford BioMedica, Genethon, Bluebird biInc.)

Youre able to thoroughly measure the competitions weaknesses and strengths together with our competitive analysis. The report, we have used total production and dispatch analysis in point of origin. Additionally, youre advised about the latest Gene Therapy industry advancements that will help you stay ahead of the competition. Our analysts are always on the feet to always track and analyze developments or changes in the Gene Therapy market. The analysis is full of statistical demonstrations and market statistics associated with sales, volume, CAGR, and share and regional and global predictions.

Gasoline Electric Solar

Rare Diseases Neurological Disorders Oncological Disorders Cardiovascular Diseases Infectious disease Other

The report supplies how big the Gene Therapy market will be in 2026 considering the studys base year 2019 and 2020. The market dynamics predominant in North America, Europe, Asia Pacific, Middle East, and Africa, and Latin America were taken into consideration and estimating the rise of the worldwide sector.

Key Questions Answered in this Report:

Futuristic Reports

Name: Alex CubbinsTel: +1-408-520-9037Email: [emailprotected]

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Gene Therapy Market Regions, Type and Application, Futuristic Study - Factory Gate

Merck is dipping its toes into a cell therapy partnership with A2 Biotherapeutics, with an offer to co-fund clinical development and allogeneic manufacturing activities through Phase I.

In particular, the pharma giant has its eyes on an undisclosed candidate utilizing A2s Tmod platform, which combines activation and blocking mechanisms in order to kill tumor cells while sparing healthy ones.

The deal features an upfront, an equity investment and reimbursement of certain expenses. Merck is also promising opt-in and milestone payments plus royalties, while keeping the door open for collaboration on a separate program.

A2 Biotherapeutics, which closed $71.5 million in Series B funding in October, has two other programs that are further along in the lead optimization phase.

The new pact brings Mercks immunotherapy and other expertise for the Tmod candidate especially in the later stages of development, manufacturing and commercialization and enables A2 to build allogeneic product development and manufacturing capabilities, said Scott Foraker, A2s president and CEO. Amber Tong

Small rare diseases biotech Soligenix $SNGX did not have a good Tuesday.

The Princeton, NJ-based company posted a Phase III fail in head and neck cancer, saying its SGX942 program did not produce a statistically significant outcome. Soligenix had been looking to treat severely inflamed mucous membranes resulting from other cancer therapies, but came up short.

Tuesdays news crushed the companys stock price, as shares were sliced by more than half at a 54% drop as the market opened. The price rebounded slightly by the end of the day, but still resulted in a 49% loss.

Soligenix did not report a p-value from the primary endpoint, which was the median duration of severe oral mucositis. They did note the data showed a 56% reduction compared to placebo, as the median in the control arm came in at 18 days and 8 days in the treatment arm.

The company tried to shine a light on a positive secondary endpoint 50% reduction in the duration of SOM in the per-protocol population. Soligenixs p-value came in here at 0.049, just clearing the statistically significant hurdle. The biotech said this endpoint may point to some evidence of biological activity.

The study enrolled 268 patients randomized 1:1 to receive either SGX942 or placebo. Soligenix said it will turn its attention toward another program, SGX301, in the treatment of cutaneous T cell lymphoma. Max Gelman

Tapping a new source for new gene therapy programs, BridgeBio has set up a three-year alliance with the University of California, San Francisco to identify early translational research that it can accelerate into the clinic.

BridgeBio, which is based in the Bay Area, said the deal follows a six-month pilot and is designed to formalize collaborative relationships with academic scientists.

The collaborations may initially take the form of sponsored research agreements with certain labs, it added, which may then lead to creation of new affiliate companies under the BridgeBio portfolio.

That pipeline currently lists three programs, utilizing AAV vectors to deliver corrective genes for congenital adrenal hyperplasia, Canavan disease and nonsyndromic hearing loss, respectively.

They are ready for more. Earlier this year, BridgeBio inked an agreement with Catalent to secure dedicated gene therapy development and manufacturing capacity to support its needs down the line. Amber Tong

Gritstone Oncology $GRTS has some new cash to play with.

The Emeryville, CA-based biotech announced Wednesday morning it had raised $110 million in private placement funding. Wednesdays deal valued company shares at $3.34 apiece, or Tuesdays closing price, and Gritstone said the funding would be primarily directed toward its GRANITE and SLATE pipeline candidates, two cancer immunotherapies.

News of the funding was met with cheers by investors, as Gritstones stock was up more than 13% in early Wednesday trading.

GRANITE, a personalized neoantigen-based immunotherapy, is being evaluated in combination studies in the Phase II portion of a Phase I/II study for microsatellite stable colorectal cancer. SLATE is also neoantigen-based and uses the same delivery system as GRANITE, but contains a fixed set of antigens rather than personalized. Its also being looked at in combination studies at the Phase II portion of a Phase I/II trial for NSCLC.

The financing was led by existing and new investors, including Redmile Group, Avidity Partners and EcoR1 Capital. The deal is expected to close by Dec. 28. Max Gelman

After closing its Series A round, Epsilon Molecular Engineering opened it back up, bagging 570 million ($5.5 million) total from investors, two bank loans and leasing.

The Saitama University spinout will use the funds for its work with heavy chain single domain antibodies, including collaborative research with Kitasato University and Kao Corporation on potential Covid-19 treatments.

EME aims to discover medium sized molecular bio-drugs with new modalities using its proprietary VHH technology, president Naoto Nemoto said in a statement. We will leverage this financing to accelerate collaborative research with pharmaceutical manufacturers and internal research using its own pipeline.

The round was led by Mitsubishi UFJ Capital, with help from Gunma Medical Engineering Vitalization Investments, Gunma Bank and Kao Corporation. EME also went forward with a subordinated loan from Shoko Chukin Bank, a loan from Saitama Resona Bank, and a lease from Syutoken Leasing. Nicole DeFeudis

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News briefing: Merck buys into A2's T cell therapy platform; Small Soligenix reports PhIII fail in head and neck cancer - Endpoints News

Last call for raising funds beforeChristmas, andthese companies didnt hesitate to scoop up their bags of cash.

TG Therapeutics

TG has been on the Nasdaq for over sevenyears now, but 2020 has been theyear for the New Yorkbiopharmato soar.With shares trading atless than $11 apiece in January, the stock is now well over $50 since theannouncementof positive topline results from two global Phase III trials for relapsing forms of MS.With a high probability of FDA approval, TG cashed inwith an upsized common stock offering,raising$300 millionto further develop and commercializetheir therapies.

BioAtla

This San Diego company hopped on the bandwagon of biopharma IPOs last week, offering 10.5M million shares at $18 apiece, a 38% increase, scooping up$189 millionin proceeds.BioAtlais developing a novel class of specific and selective antibody-based therapeutics.The companys conditionally active biologics only activate when they detect proximity to a tumor, thereby reducingsystemictoxicity.Funds will propel the companys two lead programs through three Phase II trials.

Cullinan Oncology

Breaking down the silos of drug research, Cullinan applies open innovation and collaboration to developa portfolio of first-in-class and best-in-classcancer therapies. With an oversubscribed$131.2 millionSeries C, the Cambridge company can advanceitsseven-candidatepipelineinto the clinic.Each candidate isstructured as a separate company managed by Cullinan. Two are currently in Phase I with an inhibitor drug for NSCLC and amonoclonal antibody reinvigorating the MICA/NKG2D axis.

Neurogene

New York-basedNeurogeneis establishing itself as a leader in gene therapies for neurological diseases. Last weeks$115 millionSeries B round willhelp advance multiple of the companys candidates into the clinic. The first of which targetslate infantileBattens Disease,a rare nervous system disorder that worsens over time and is fatal, usually 8-10 years old.The funds will also be used to build outNeurogenesadeno-associated virus vector GMP manufacturing capabilities.

Neuron23

Having worked undercover for two years, Neuron23 uncloakedlast weekwith$113.5 millionin financing for its launch.$30 million of the funds came from Westlake Village BioPartners, who just announcedtwo funds totaling$500 millionwith the intent to invest in Series A startups in the most promising companies.Neuron23 has hit the ground running, aiming to take on Parkinsons diseaseagainst giant Biogen, who recentlyorchestratedin a $1 billion dealwith Denaliwith the same target in mind. The plan is to start trials with healthy volunteers next year.

Neomorph

Established earlier this year, San Diego-based Neomorph raked in$109 millionin a Series A. The companys focus is on targeted proteindegradation, which offers opportunities for treatment developments across the board, including oncology.The Neomorph team has deep expertise in pharmacological approaches to targeted protein degradation and we are excited to be developing new therapeutics for patients with diseases that are currently difficult to treat, said scientific founderScottArmstrongMD, Professor of Pediatrics at Harvard Medical School and the Dana-Farber Cancer Institute.

AtsenaTherapeutics

Gene therapy startupAtsenaclosed on an oversubscribed$55 millionSeries A.The funds will be used to advance itsgene therapy for one of the most common cause of blindness in children through clinical trials.Leber congenital amaurosis (LCA) causes blindness in 2 to 3 out of 100,000 newborns.The company isalso planning for growth,looking to move into a larger space next yearto scale up gene therapy manufacturing. Ramping up across the board, theres a goal to hire 20 more positions with the move.

ONL Therapeutics

With support from Johnson & Johnson and more, Michigan-based ONL closed a$46.9 millionSeries B Preferred Stock financing round.The company is developing therapies for protecting the patients with retinal disease from vision loss.This funding supports the completion of a Phase 1 study in retinal detachment with ONLs lead compound ONL1204. In addition, the funding will advance ONL1204 in two chronic indications, glaucoma and dry age-related macular degeneration.

Peptilogics

Peptilogicsis the most recent biotech receiving investments fromPaypalsco-founder Peter Thiel. This week, Thiel participated in a$35 millionfinancing round for the Pennsylvania-basedcompany.The funds will be used to advancePeptilogicsproprietarycomputational peptide drug design and discovery platform. The platform discovers connections in diverse biomedical data and maps peptide sequences.

Octave Bioscience

Looking totakeitsfully integratedcare management platform to the next level, Octave completed a$32 millionSeries B financing round. The funds will allow thecompany to complete development of management products and services, expand clinical data and begin commercialization to neurologists and patients. Octaves first target has been multiple sclerosis patients, but will expand to other chronic, debilitating neurodegenerative diseases. Theplatformtracksblood-basedbiomarkers, enhanced MRI insights and mobile patient monitoring tools to feed intocare pathwaymodels to generatebetter patient outcomes and lower costs.

Vivace Therapeutics

Small molecule player Vivace closed a$30 millionSeries C for further development of its first-in-class therapies targeting the Hippo pathway. Funds will be usedto take its lead candidate into first-in-human studies in early 2021, targeting tumors dependent on activated YAP.Pre-clinical research has shown promise for the candidate both as a monotherapy and in combination with other anti-cancer therapies.

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MALVERN, Pa., Dec. 23, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc., (NASDAQ: OCGN), a leading biopharmaceutical company focused on discovering, developing and commercializing a pipeline of innovative therapies, today announced the appointment of a vaccine scientific advisory board comprised of leading academic and industry experts in the vaccine field to evaluate the clinical and regulatory path to approval in the US market of Bharat Biotechs COVAXIN, a whole-virion inactivated COVID-19 vaccine candidateto be co-developed by Ocugen and Bharat Biotech for the US market.

Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen remarked, We are thrilled to welcome this group of esteemed thought leaders to the Ocugen team to assist in our co-development with Bharat Biotech of COVAXIN. This unique yet traditional vaccine candidate is different from other options currently available in the US market with potentially broader coverage against multiple protein antigens of the virus.

The vaccine scientific advisory board consists of:

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. For more information, please visit http://www.ocugen.com.

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Ocugen Contact:Ocugen, Inc.Sanjay SubramanianChief Financial Officerir@ocugen.com

Media Contact:LaVoieHealthScienceLisa DeScenzaldescenza@lavoiehealthscience.com+1 978-395-5970

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Advances Eden BioCells clinical program to validate Rapid Personalized Manufacturing (RPM)

Clinical trial to study autologous CD19-specific CAR-T using RPM technology designed to reduce cost and simplify production for infusion the day after gene transfer

BOSTON, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Ziopharm Oncology, Inc. (Ziopharm or the Company) (Nasdaq: ZIOP), today announced that the Taiwan Food and Drug Administration has cleared an investigational new drug application (IND) from Eden BioCell, a joint venture between Ziopharm and cell therapy company TriArm Therapeutics, for its phase 1 clinical trial to evaluate patient-derived CD19-specific CAR-T, using Ziopharms Rapid Personalized Manufacturing (RPM) technology. This is an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas and the first clinical study of autologous non-viral CD19-specific CAR-T in Taiwan.

This trial will utilize Ziopharms non-viral Sleeping Beauty cell engineering technology to infuse autologous CAR-T the day after T cells have been genetically modified. Ziopharms RPM CD19-specific CAR-T therapy results from the stable, non-viral insertion of DNA into the genome of resting T cells to co-express the chimeric antigen receptor (CAR), membrane-bound IL-15 (mbIL15) and a safety switch. The trial is being conducted at National Taiwan University Hospital.

This study is a testament to the relationship Ziopharm has quickly established with Eden BioCell and TriArm and the progress using patients T cells under RPM to target malignancies, said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. The results will help us understand the benefit of engineering T cells with membrane bound IL-15 which could benefit not only CAR-T, but also the engineering of T cells to express T-cell receptors.

Jay Zhang, Co-Founder and Chief Executive Officer of TriArm, added, We are very excited to receive clearance of our IND in Taiwan. The learnings from this study will build upon the encouraging early data we are seeing with patients treated with RPM CAR-T targeting CD19 malignancies under compassionate use. We believe our approach has the potential to transform CAR-T therapy by dramatically decreasing the amount of time needed for manufacturing engineered T cells, thereby increasing efficacy and decreasing cost.

CAR-T therapy has proved an effective therapy for B-cell cancers, noted Dr. Shang-Ju Wu, Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital and Principal Investigator for the study. Further optimization by shortening the manufacturing time would be of great importance to make this therapy more available to patients. We are honored to be involved in the clinical development of this non-viral CAR-T therapy produced using RPM. We hope the data derived from this current trial will advance CAR-T therapy to benefit our patients.

Up to 24 patients with relapsed CD19+ leukemias and lymphomas will be enrolled in this phase 1 trial, with the goal of infusing 16 subjects (Taiwan FDA #1096030182). The primary endpoint of the study is to evaluate the safety and tolerability of autologous CD19-specific T cells manufactured using the RPM process.

About Eden BioCell In December 2018, Ziopharm and TriArm Therapeutics announced the launch of Eden BioCell to lead clinical development and commercialization of Sleeping Beauty-generated CAR-T therapies in Greater China. Ziopharm licensed the rights to Sleeping Beauty-generated CAR-T therapies targeting the CD19 antigen using Ziopharms RPM technology in Greater China to Eden BioCell. TriArm has committed up to $35 million to this joint venture, and Eden BioCell is owned 50-50 by Ziopharm and TriArm.

About TriArm TherapeuticsTriArm Therapeutics is a cell therapy company formed by Panacea Venture with R&D operations in Germany, United States and Greater China region. The company is dedicated to the treatment of cancer and autoimmune diseases.

About Ziopharm Oncology, Inc.Ziopharm is developing non-viral and cytokine-driven cell and gene therapies that weaponize the bodys immune system to treat the millions of people globally diagnosed with cancer each year. With its multiplatform approach, Ziopharm is at the forefront of immuno-oncology. Ziopharms pipeline is built for commercially scalable, cost effective T-cell receptor T-cell therapies based on its non-viral Sleeping Beauty gene transfer platform, a precisely controlled IL-12 gene therapy, and rapidly manufactured Sleeping Beauty-enabled CD19-specific CAR-T program. The Company has clinical and strategic collaborations with the National Cancer Institute, The University of Texas MD Anderson Cancer Center and Regeneron Pharmaceuticals. For more information, please visit http://www.ziopharm.com.

Forward-Looking Statements DisclaimerThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the potential benefits of the Companys CAR-T therapy and the Companys expectations regarding the number of patients expected in this phase 1 clinical trial. Although Ziopharms management team believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Ziopharm, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, changes in Eden BioCells operating plans that may impact its cash expenditures, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Ziopharms product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies and for which indication; the strength and enforceability of Ziopharms intellectual property rights; competition from other pharmaceutical and biotechnology companies as well as risk factors discussed or identified in the public filings with the Securities and Exchange Commission made by Ziopharm, including those risks and uncertainties listed in Ziopharms Quarterly Report on Form 10-Q filed by Ziopharm with the Securities and Exchange Commission. We are providing this information as of the date of this press release, and Ziopharm does not undertake any obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason.

Investor Relations Contacts:Adam D. Levy, PhD, MBAEVP, Investor Relations and Corporate CommunicationsT: 508.552.9255E: alevy@ziopharm.com

Media Relations Contact:LifeSci Communications:Patrick BurseyT: 646.876.4932E: pbursey@lifescicomms.com

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Ziopharm Oncology Announces Clearance of Taiwan's First IND of Non-viral CAR-T for the Treatment of Relapsed CD19+ Leukemias and Lymphomas -...

SOUTH SAN FRANCISCO, Calif., Dec. 23, 2020 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer today announced that theU.S. Food & Drug Administration(FDA) has cleared an Investigational New Drug (IND) application to study ALLO-715, Allogenes investigational BCMA AlloCAR T therapy, in combination with nirogacestat, SpringWorks Therapeutics investigational gamma secretase inhibitor (GSI), in patients with relapsed or refractory multiple myeloma. This combination is part of the companys multi-pronged strategy to develop a treatment for multiple myeloma and will be deployed in the ongoing UNIVERSAL trial. Enrollment in this cohort is expected to begin in the first quarter of 2021.

We are delighted that the FDA has cleared our IND application for ALLO-715 in combination with nirogacestat, said Rafael Amado, M.D., Executive Vice President of Research & Development and Chief Medical Officer of Allogene. We look forward to investigating this combination as part of our comprehensive anti-BCMA strategy aimed at optimizing cell therapy for patients with relapsed/refractory multiple myeloma.

Gamma secretase is an enzyme that cleaves BCMA from the surface of myeloma cells. In preclinical models, nirogacestat has been shown to prevent the cleavage and shedding of BCMA, leading to an increase in the cell surface density of BCMA and reduced levels of soluble BCMA.1 Increasing BCMA surface expression with gamma secretase inhibitor may enable deeper and more durable responses to ALLO-715 in patients with multiple myeloma.

Multiple myeloma is the second most common hematological malignancy in the United States, with 32,270 new cases and 12,830 deaths estimated in 2020.2

The Phase 1 combination trial is being advanced pursuant to a clinical trial collaboration agreement that Allogene and SpringWorks entered into in January 2020. Under the terms of the agreement, Allogene is sponsoring and conducting the Phase 1 study to evaluate the safety, tolerability and preliminary efficacy of the combination, and is assuming all development costs associated with the study, other than expenses related to the manufacturing of nirogacestat and certain expenses related to intellectual property rights. Allogene and SpringWorks have formed a joint development committee to oversee the clinical study.

About ALLO-715ALLO-715, an AlloCAR T therapy targeting B-cell maturation antigen (BCMA), is a potential novel treatment for multiple myeloma and other BCMA-positive malignancies. Multiple myeloma originates in the bone marrow and it is characterized by abnormalities in plasma cells that reproduce uncontrollably in the bone marrow and other disease sites.3 Multiple myeloma is incurable for most patients, as relapses occur despite most treatments available.4 Initial results from the Phase 1 UNIVERSAL study of ALLO-715 in relapsed/refractory multiple myeloma were presented at an oral session of the American Society of Hematology (ASH) annual meeting in December 2020. This study also uses ALLO-647, Allogene's anti-CD52 monoclonal antibody (mAb), as a part of its differentiated lymphodepletion regimen.

ALLO-715 utilizes the TALEN gene-editing technology pioneered and owned by Cellectis. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed at the BCMA target. Allogene holds the global development and commercial rights for this investigational candidate.

About NirogacestatNirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often recurrent, debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy binding molecules for BCMA-directed therapies. Nirogacestats ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma.

Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

AboutAllogene TherapeuticsAllogene Therapeutics, with headquarters inSouth San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T) therapies for cancer. Led by a management team with significant experience in cell therapy, Allogene is developing a pipeline of off-the-shelf CAR T cell therapy candidates with the goal of delivering readily available cell therapy on-demand, more reliably, and at greater scale to more patients. For more information, please visitwww.allogene.com, and follow @AllogeneTx on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the ability and timing to initiate a clinical trial of ALLO-715 in combination with nirogacestat; ability to manufacture ALLO-715; the ability of ALLO-715 in combination with nirogacestat to enable deeper or more durable responses; and the potential benefits of AlloCAR T therapy. Various factors may cause differences between Allogenes expectations and actual results as discussed in greater detail in Allogenes filings with the SEC, including without limitation in its Form 10-Q for the quarter ended September 30, 2020. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

AlloCAR T is a trademark of Allogene Therapeutics, Inc.

Allogene Media/Investor Contact:Christine CassianoChief Communications Officer(714) 552-0326Christine.Cassiano@allogene.com

1 Eastman S, Shelton C, Gupta I, Krueger J, Blackwell C, Bojczuk. Synergistic activity of belantamab mafodotin (anti-BCMA immuno-conjugate) with PF-03084014 (gamma-secretase inhibitor) in Bcma-expressing cancer cell lines. Blood. 2019;134(supplement_1):4401. doi.org/10.1182/blood-2019-123705.2 https://www.cancer.org/cancer/multiple-myeloma/about/what-is-multiple-myeloma.html3 Multiple myeloma - Genetics Home Reference - NIH. Retrieved from https://ghr.nlm.nih.gov/condition/multiple-myeloma#4 Sonneveld P, Broijl A. Treatment of relapsed and refractory multiple myeloma. Haematologica. 2016;101(4):396-406

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Allogene Therapeutics Receives IND Clearance from the U.S. Food and Drug Administration for ALLO-715 in Combination with Nirogacestat in...

LONDON--(BUSINESS WIRE)--Gyroscope Therapeutics Limited, a clinical-stage gene therapy company focused on diseases of the eye, today announced the company has entered a sponsored research agreement with the University of Pennsylvania and the Penn Center for Advanced Retinal and Ocular Therapeutics (CAROT) to develop gene therapies for serious eye diseases that can lead to permanent vision loss. Gyroscope has an exclusive option to the intellectual property associated with, and arising from, the research conducted under the agreement.

A team of researchers from CAROT and Gyroscope will work together to explore specific gene therapy targets for glaucoma, optic neuritis and retinitis pigmentosa. The CAROT team is led by Jean Bennett, M.D., Ph.D., the F.M. Kirby Professor of Ophthalmology, along with Ken Shindler, M.D., Ph.D., an Associate Professor of Ophthalmology and Ahmara Ross, M.D., Ph.D., an Assistant Professor of Ophthalmology, of the Perelman School of Medicine.

Too many people around the globe face a life with limited vision or complete blindness because current treatment options for many serious eye diseases are so limited, said Khurem Farooq, Chief Executive Officer, Gyroscope. Gene therapy has the potential to be a completely new way of approaching these diseases, and we are very excited to work with Jean and the team of world leaders in ophthalmic gene therapy research at the University of Pennsylvania to evaluate new targets for these conditions.

Our team is passionate about the potential of gene therapies for people with serious eye diseases, said Dr. Bennett. We are looking forward to furthering our research in glaucoma, optic neuritis and retinitis pigmentosa, which combined currently cause a devastating loss of vision for millions of people around the world.

Glaucoma is a leading cause of irreversible blindness globally. An estimated 80 million people have glaucoma worldwide, and this number is expected to increase to more than 111 million by 2040.1 There is no cure for glaucoma. If it is caught early, people with glaucoma can be treated with surgery or medication to help control the disease. Because glaucoma typically does not cause pain, it often progresses silently and is not diagnosed until the optic nerve is irreparably damaged.

Retinitis pigmentosa (RP) refers to a group of rare genetic retinal diseases that cause progressive loss of night and peripheral vision. The condition is often diagnosed in childhood or adolescence and can lead to legal, and sometimes complete, blindness. An estimated 300,000 people worldwide have RP, mainly caused by a genetic variant inherited from one or both parents.2

Optic neuritis occurs when the optic nerve is damaged as a result of inflammation. Symptoms of optic neuritis include temporary vision loss in one eye and pain with eye movement. Optic neuritis is closely associated with multiple sclerosis (MS): It is the first sign of MS in 20% of patients and occurs during the course of the disease in 50% of MS patients.3

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage gene therapy company developing gene therapy beyond rare disease to treat diseases of the eye that cause vision loss and blindness. Our lead investigational gene therapy, GT005, is currently being evaluated in Phase II clinical trials for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), a leading cause of blindness. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 has received Fast Track designation from the U.S. Food and Drug Administration for the treatment of people with GA.

Syncona Ltd, our lead investor, helped us create a leading gene therapy company combining discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

1 Tham YC, Li X, Wong TY, Quigley HA, Aung T, Cheng CY. Global prevalence of glaucoma and projections of glaucoma burden through 2040: a systematic review and meta-analysis. Ophthalmology. 2014 Nov;121(11):2081-90.

2 Cowen Equity Research Therapeutic Categories Outlook: Comprehensive Study. 2020 Feb;P.2334.

3 Kale N. Optic neuritis as an early sign of multiple sclerosis. Eye Brain. 2016;8:195-202.

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Gyroscope Therapeutics and the University of Pennsylvania Announce Research Agreement to Develop Gene Therapies for Serious Eye Diseases - Business...

18 months into the first serious clinical trials of CRISPR gene therapy for sickle cell disease and beta-thalassemiaand all patients are free from symptoms and have not needed blood transfusions.

Sickle cell disease (SCD) can cause a variety of health problems including episodes of severe pain, called vaso-occlusive crises, as well as organ damage and strokes.

Patients with transfusion-dependent thalassemia (TDT) are dependent on blood transfusions from early childhood.

The only available cure for both diseases is a bone marrow transplant from a closely related donor, an option that is not available for the vast majority of patients because of difficulty locating matched donors, the cost, and the risk of complications.

In the studies, the researchers goal is to functionally cure the blood disorders using CRISPR/Cas9 gene-editing by increasing the production of fetal hemoglobin, which produces normal, healthy red blood cells as opposed to the misshapen cells produced by faulty hemoglobin in the bodies of individuals with the disorders.

The clinical trials involve collecting stem cells from the patients. Researchers edit the stem cells using CRISPR-Cas9 and infuse the gene-modified cells into the patients. Patients remain in the hospital for approximately one month following the infusion.

Prior to receiving their modified cells, the seven patients with beta thalassemia required blood transfusions approximately every three to four weeks and the three patients with SCD suffered episodes of severe pain roughly every other month.

All the individuals with beta thalassemia have been transfusion independent since receiving the treatment, a period ranging between two and 18 months.

Similarly, none of the individuals with SCD have experienced vaso-occlusive crises since CTX001 infusion. All patients showed a substantial and sustained increase in the production of fetal hemoglobin.

15 months on, and the first patient to receive the treatment for SCD, Victoria Gray, has even been on a plane for the first time.

Before receiving CRISPR gene therapy, Gray worried that the altitude change would cause an excruciating pain attack while flying. Now she no longer worries about such things.

She told NPR of her trip to Washington, D.C: It was one of those things I was waiting to get a chance to do It was exciting. I had a window. And I got to look out the window and see the clouds and everything.

MORE: MIT Researchers Believe Theyve Developed a New Treatment for Easing the Passage of Kidney Stones

This December, theNew England Journal of Medicinepublishedthe first peer-reviewed research paperfrom the studyit focuses on Gray and the first TDT patient who was treated with an infusion of billions of edited cells into their body.

There is a great need to find new therapies for beta thalassemia and sickle cell disease, saidHaydar Frangoul, MD,Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center. What we have been able to do through this study is a tremendous achievement. By gene editing the patients own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases.

READ: For the First Time in the US, Surgeons Pump New Life into Dead Donor Heart for Life-Saving Transplant

Because of the precise way CRISPR-Cas9 gene editing works, Dr. Frangoul suggested the technique could potentially cure or ameliorate a variety of diseases that have genetic origins.

As GNN has reported, researchers are already using CRISPR to try and treat cancer, Parkinsons, heart disease, and HIV, as well.

Source: American Society of Hematology

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Every Patient Treated With CRISPR Gene Therapy for Blood Diseases Continues to Thrive, More Than a Year On - Good News Network

When gene therapy developer Generation Bio raised $110 million in venture funding in January and then followed up six months later with a $230 million initial public offering, it was as sure asign as any that investors are stoked about the next generation of gene therapies to treat rare diseases.

Their enthusiasm hasnt waned during the year, either, despite challenges ranging from the COVID-19 pandemic delaying clinical trials to regulators pushing back some development timelines so they can gather more data on emerging gene therapies.

And as two FDA-approved gene therapies for rare diseases gain ground in the marketSpark Therapeuticss Luxturna for RPE65 mutation-associated retinal dystrophy and Novartis Zolgensma for spinal muscular atrophy (SMA)the biopharma industry is hard at work on novel approaches to correcting rare disorders caused by errant genes. The advances range from new gene-insertion methods to innovations that allow the therapies to penetrate hard-to-reach tissues in the body.

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Some, like Generation, are directly addressing one big concern that has plagued the first generation of gene therapies: Just how durable are they? Its a question BioMarin faced in August when the FDA declined to approve its hemophilia A gene therapy valoctocogene roxaparvovec after data from a trial showed that levels of factor VIII fell 12 to 18 months after patients received the gene therapy, which is designed to restore the critical blood-clotting protein.

Generations lead gene therapy candidates are designed to treat rare blood disorders hemophilia A and phenylketonuria (PKU), and theyre still in preclinical development. Whats new about the company's approachis the delivery system: Rather than using a virus to insert a gene correction, Generation Biouses an alternative technology that avoids touching off an immune responsea buildup of antibodies to the virus that would normally prevent a second round of treatment.

Generations core technology, called non-viral closed-ended DNA (ceDNA), is carried into the body by a lipid nanoparticle. The potential for the technology to sidestep the immune response thats typical with virus-based gene therapies could be important in diseases like PKU, where the gene correction needs to reach liver cells, or hepatocytes.

The newborn liver divides incredibly quickly, and as it grows, the dose of gene therapy goes down, said Geoff McDonough, M.D., CEO of Generation Bio, in an interview. We dont view that as an existential problem. Well just re-dose.

BioMarin, meanwhile, is working with the FDA to address its request for more data on valoctocogene roxaparvovec, which is an adeno-associated virus (AAV)-based gene therapybut its also looking ahead to innovations that could improve future iterations of the technology. For one thing, it'sinvestigating different capsids that that may reduce the immune response to the first dose, thus allowing re-dosing later.

But that may be only a small part of addressing a decline in response to gene therapy. We also have to understand cellular determinants of expression, because maybe re-dosing isnt actually the answer after all, said Hank Fuchs, M.D., president of research and development at BioMarin, in an interview. To that end, BioMarin is studying liver biopsy tissueto try to understand how individual characteristics may affect the fate of the transgene.

And BioMarin is working with Swiss startup Dinaqor to develop gene therapies to treat heart diseases such as hypertrophic cardiomyopathy. To accomplish that, the companies are making capsids that travel not to the liverthe destination of many gene therapiesbut to the heart. If they succeed, it could be a significant platform play for us, Fuchs said. The morbidity for hypertrophic cardiomyopathy is terrible and 60% of cases are genetic. If we can do cardiac delivery, there are other genetic diseases that could be treated with gene therapy.

In 2019, a group of executives who had pioneered SMA gene therapy Zolgensma launched Taysha Gene Therapies with an ambitious goal: They wanted to correct genetic nervous system disorders by delivering gene therapies directly to the spinal fluid. Now, backed by $125 million in private funding and a $157 million IPO, Taysha is in preclinical testing withthree gene therapies for neurodegenerative diseases.

Tayshas gene therapy for GM2 gangliosidosis, a disease that progressively destroys nerve cells, is distinctive for more than its intrathecal delivery, said CEO RA Session II in an interview.

The therapy uses a single viral vector to deliver not one, but two genes at the heart of the disorderHEXA and HEXB. Theyre linked by a self-cleaving peptide and a promoter, which allows the two genes to be expressed at a one-to-one ratio, mimicking the endogenous system of a healthy cell, Session explained in an interview.

Other gene therapy developers are targeting specific cells in the body with new technology. Encoded Therapeutics, for example, is developing a gene therapy to treat the seizure disorder Dravet syndrome. But rather than replacing the mutated SCN1A gene that causes the disorder, Encoded incorporates pieces of DNA into an AAV vector with the goal of dialing up production of the SCN1A protein thats needed to correct the disorder.

RELATED: Encoded Therapeutics bags $135M to push 'precision gene therapy' into the clinic

Passage Bio is addressing GM1 gangliosidosis using a next-generation viral vector called AAVhu68, which in preclinical trials increased the expression of a needed protein not only in targeted cells, but also in the cerebral spinal fluid. The protein is then taken up by neighboring cells, creating an effect of cross correction that the companys scientists hope will improve developmental milestones and survival in the children who have the disease, said CEO Bruce Goldsmith, Ph.D., in an interview.

In August, Passage Bios planned phase 1/2 trial was placed on a clinical hold by the FDA, which cited concerns about the delivery device planned for the trial. The company is conducting risk assessments and testing the device so it can address the agencys questions, and Goldsmith expects to maintain a close dialogue with the FDA going forward.

Infantile GM1 can occur quite early, so we want to make sure the FDA is a collaborator on defining what developmental scales will be appropriate for measuring outcomes. That means not only primary outcomes but also durabilitywhat theyre looking for in terms of meaningful outcomes, he said. U.K. regulators gave their go-ahead for a clinical trial of the therapy in December.

Improving cross-correction in gene therapy is also a priority for Avrobio, which is developing gene therapies for several rare diseases, including Hunter syndrome and Fabry disease. Its technology platform, called plato, consists of a lentiviral vector and tags that help the therapeutic proteins reach the target cells lysosomesthe organelles inside of cells that orchestrate vital processes in the body.

In diseases like Fabry, all thats needed is cross-correction, where the enzyme in circulation is taken up by the cells and creates a profound effect, correcting a deficiency that causes organ damage, said CEO Geoff MacKay in an interview.The tags aid the uptake of a therapeutic protein. Its like a first-class ticket to the target tissues, like muscles and the central nervous system."

In November, Avrobio announced that in phase 1 and 2 trials of its Fabry genetherapy, the response lasted up to 3.5 years.

RELATED: Avrobio tracks improvements in first patient treated with Gaucher gene therapy

LogicBio Therapeutics approach to moving gene therapy into the future is to harness the power of genome editing.

The companys technology, GeneRide, uses strands of DNA to deliver a functioning copy of a faulty gene into cells nuclei, prompting natural DNA repair mechanisms to insert the good gene exactly where it belongs in the chromosome. The therapeutic gene becomes part of that celland of its daughter cells when it dividespotentially preventing a dilution of effect over time that can occur with other gene therapies.

LogicBios lead program, LB-001 to treat the liver disorder methylmalonic acidemia in children age 3 and older, was hit with a delay in February, when the FDA put a hold on the planned clinical trial so the company could address safety-monitoring concerns.

So LogicBio built in a protocol for caregivers to monitor post-treatment safety at home, and it added survival as a secondary endpoint, said LogicBios chief operating officer Kyle Chiang, Ph.D., in an interview. The company hopes to dose the first patient in the trial in early 2021.

BioMarins Fuchs predicts that each new development in gene therapy will raise more questions for the FDAbut that the delays wont prevent the advances from benefiting patients.

As regulators, its not in their DNA to take risks, Fuchs said. But the quest for gene therapy approvals, he added, will continue to go well, as regulators get more familiar with the technology and developers generate more and more data.

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The next generation of gene therapy for rare diseases forges ahead as developers weather hurdles - FierceBiotech

Round was led by Sofinnova Investments with participation from Abingworth, Lightstone Ventures and all existing investors

Company expands board of directors and plans to build out team

DURHAM, N.C. and BOSTON, Dec. 16, 2020 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced it has closed an oversubscribed $55 million Series A financing led by Sofinnova Investments with participation from additional new investors Abingworth and Lightstone Ventures. Founding investors Hatteras Venture Partners and the Foundation Fighting Blindness RD Fund, along with existing investors Osage University Partners, University of Florida, and Manning Family Foundation, also participated in the round. Sarah Bhagat, PhD, Partner at Sofinnova, Jackie Grant, PhD, Principal at Abingworth, and Jason Lettmann, General Partner at Lightstone, will join Atsenas board of directors.

Proceeds will be used to advance Atsenas ongoing Phase I/II clinical trial evaluating a gene therapy for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), one of the most common causes of blindness in children, as well as complete manufacturing development for Phase 3. In addition, the funds will enable Atsena to expand its team to support the research and development of novel gene therapies, including the progression of two existing preclinical programs in inherited retinal diseases toward the clinic and advancement of the companys innovative adeno-associated virus (AAV) technology platform.

We are grateful for the support of our new and existing investors and are encouraged by their enthusiasm for the potential of our technology to overcome the unique hurdles of inherited retinal diseases to prevent or reverse blindness, said Patrick Ritschel, MBA, Chief Executive Officer of Atsena. The Series A financing provides financial runway to reach the key inflection point of reading out efficacy data from our LCA1 clinical trial. While we continue expeditiously advancing this trial and our preclinical programs, we are excited to accelerate our growth as a leading ophthalmic gene therapy company.

The Phase I/II LCA1 clinical trial is currently enrolling patients in the second dosing cohort. Atsena exclusively licensed the rights to the gene therapy from Sanofi, which originally licensed it from University of Florida. LCA is the most common cause of blindness in children. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this inherited retinal disease.

We believe Atsenas foundation in ocular gene therapy and potentially game-changing novel AAV vectors position the company to become a partner of choice, said Dr. Bhagat. Sofinnova is delighted to support Atsena and we look forward to helping the team further its mission to develop life-changing gene therapies for patients with inherited retinal diseases.

About Atsena TherapeuticsAtsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The companys ongoing Phase I/II clinical trial is evaluating a potential therapy for one of the most common causes of blindness in children. Its additional pipeline of leading preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome significant hurdles presented by inherited retinal disease, and its unique approach is guided by the specific needs of each patient condition to optimize treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye, Atsena has a licensing, research and manufacturing collaboration with the University of Florida and has offices in Boston, MA and North Carolinas Research Triangle, environments rich in gene therapy expertise. For more information, please visit atsenatx.com.

About Sofinnova InvestmentsSince our founding in 1974, Sofinnova has been active in life science investing. We are a clinical-stage biopharmaceutical investment firm with approximately $2.3B in assets under management and committed capital. We invest in both private and public equity of therapeutics-focused companies. Our goal is to actively partner with entrepreneurs in both the U.S. and Europe, across all stages of company formation. From drug development and navigating the regulatory process to company building and IPO, we strive to be collaborative, meaningful board members, and excellent partners at every level. We seek to build world class companies that aspire to dramatically improve the current state of medical care today and ultimately, the lives of patients. Sofinnova has expertise investing in gene therapy companies, including investments in Spark, which developed the first approved gene therapy, Akouos, and Audentes, and Xylocor. For more information, please visit http://www.sofinnova.com.

About Abingworth Abingworth is a leading transatlantic life sciences investment firm. Abingworth helps transform cutting-edge science into novel medicines by providing capital and expertise to top caliber management teams building world-class companies. Since 1973, Abingworth has invested in approximately 168 life science companies, leading to more than 44 M&A/exits and close to 70 IPOs. Our therapeutic focused investments fall into 3 categories: seed and early-stage, development stage, and clinical co-development. Abingworth supports its portfolio companies with a team of experienced professionals at offices in London, Menlo Park (California) and Boston. For more information, visit abingworth.com.

About Lightstone VenturesLightstone Ventures is a leading venture capital firm investing in therapeutic-oriented companies across the life science spectrum, from breakthrough medical devices to novel drugs and biopharmaceuticals. Founded in 2012, Lightstone has been part of many successful new ventures from inception through commercialization and plays a critical role guiding and building successful healthcare companies. With a proven strategy and global footprint, the Lightstone team has been involved in several of the largest venture-backed life science exits over the last decade including: ALX Oncology, Acceleron, Ardian, Calithera, Claret Medical, Disarm, MicroVention, Nimbus, Plexxikon, Portola, Promedior, Proteolix, Ra Pharma, Tizona, Twelve and Zeltiq. For more information, visithttps://www.lightstonevc.com.

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Atsena Therapeutics Raises $55 Million Series A Financing to Advance LCA1 Gene Therapy Clinical Program, Two Preclinical Assets, and Novel Capsid...

When did you first start using AAV vectors in your gene therapy work?

It came about 10 years ago when I was helping the Gates Foundation develop an approach for preventing HIV. Any attempt to use a traditional vaccine, where you inject a component of the virus to activate the immune system to develop proteins such as antibodies, had been challenging for HIV. Regardless of what you used to immunizebecause the virus changed so muchmost of it would escape. Once the field realized that, we started to look at other approaches, and it turns out its possible in the lab to engineer an antibody that could be effective against many types of HIV.

HIV represents a different type of pandemic than COVID-19. When did you turn to AAV vectors as a potential approach for other kinds of pandemics?

About eight years ago I started thinking about this as a countermeasure for a pandemic. The pandemics that we worry about are primarily transmitted through a respiratory route. If it were direct contact like Ebola virus, its not as dangerous because you can avoid touching one another. But if you cant even be in the same room, thats a problem.

Respiratory viruses enter our body through the nose and throat. Thats how we get infected. We proposed delivering the vector through a nasal mist or spray to engineer the cells that line the nose and throat to express the antibody. If you can localize this at that site to prevent the virus from going farther, then you dont need the whole body to express the antibodies.

The antibodies youre using, called casirivimab and imdevimab, are monoclonal antibodies, meaning they were created in a lab. Can you describe how they work?

Regeneron developed these. Theyre highly active and potent against SARS-CoV-2. For treatment, antibodies can be useful. If youre starting to get sick, you get an infusion or two of the antibodies and then you dont get sicker. But what do you do with 99% of the population who isnt sick and never gets sick? Our idea was to use an AAV vector expressing the antibodies to engineer someones cells to produce the antibodies. If we do this right, the expression could go on for a long period of time. Its a one-time vector infusion.

We were able to show in animal models that an AAV sprayed into the nose that expresses an antibody is effective against flu virus that causes respiratory diseases and has the potential to cause a pandemic. The treated animals were completely protected when exposed to flu virus. Its all about having the right antibody and then engineering a delivery system to have this blockage. We call it a bioshield. It could be a way to stop COVID-19 in its tracks.

Would this approach replace COVID-19 vaccines or be used in conjunction with them?

Theoretically, it could be used in place of a vaccine, but I suspect that traditional vaccines are going to succeed for a lot of people. We see our approach being deployed in individuals for which traditional vaccines may not work as well, patients with diseases that compromise their immune system such as cancer, patients who are on immune-modulating drugs, or even the elderly.

Early data seem to suggest that the elderly have some level of response to the active COVID-19 vaccine, but, like with many other vaccines, older people dont mount the same immune response as those who are younger. That said, I dont see any reason why receiving a traditional vaccine would preclude one from using our nasal spray because they do two different things.

The other possibility is that the COVID-19 vaccines we have become less effective because the virus changes. I dont think this will happen, and I hope it doesnt, but, if it does, the question becomes, Would the antibodies that Regeneron created become a backup? When we roll out an active vaccine based on a single spike protein into large populations, it creates pressure on the SARS-CoV-2 to change and potentially become resistant. I hope a variant doesnt emerge, but I do think it behooves us to have some redundancy in place to squelch a potential second wave due to resistant coronaviruses.

What is your projected timeline?

We are conducting one final experiment over the holiday break and in early January before we submit our request to the FDA for clinical trials. Weve had discussions with the FDA and have already done some of the initial testing, including safety testing in nonhuman primates, as well as preparing to manufacture the product. Conducting this in the Gene Therapy Program is beneficial since we are comfortable with AAV vectors and moving them into clinical trials. We support up to eight traditional AAV gene therapy programs a year, and we have the staff and technology to move pretty quickly.

If we get the go in January, I think our technology could contribute to the global response in eliminating COVID-19. And you have to understand, until we eliminate it globally, we havent actually eliminated it.

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Repurposing a proven gene therapy approach to treat, prevent COVID-19 - Penn Today

San Diego-based Locanabio secured $100 million in a Series B financing round that will be used to advance the companys portfolio of novel RNA-targeted gene therapies for neurodegenerative, neuromuscular and retinal diseases.

The funding will support pre-clinical and clinical development of its gene therapy treatments for diseases such as Huntington's disease, myotonic dystrophy type 1, genetic forms of amyotrophic lateral sclerosis and retinal disease, the company said.

Locanabio has a unique approach to gene therapy. The company has combined two validated gene therapy and RNA modifications to treat diseases. Locanabio uses a gene therapy vector to deliver an RNA-targeting protein tipped with an RNA-modifying enzyme. Through targeting RNA, the company said its approach avoids the risk of off-target effects in DNA and is suited to address many diseases linked to dysfunctional processing of RNA.

The $100 Million Series B builds on $55 million the company secured in a Series A last year. Chief Executive Officer Jim Burns, who joined Locanabio one year ago, said the financing round will allow the company to advance several of the companys most promising programs into IND-enabling studies in 2021. The financing will also allow the company to continue to advance its RNA-targeting platform, which has the potential to be a major new advance in medicine that can bring hope to patients with many devastating genetic diseases, Burns said.

While all of Locanabios assets are still in the research phase, its most advanced is a therapy for myotonic dystrophy type 1 (DM1), a genetic neuromuscular disorder caused by a mutation in the DMPK gene that results in trinucleotide (CUG) repeat expansion in the expressed RNA. Locanabios DM1 program targets and destroys the toxic CUG repeats, according to company information. Earlier this year, as BioSpace previously reported, Locanabio published a paper demonstrating the benefits of its technology as a potential one-time treatment of DM1.

The financing round was led by Vida Ventures LLC with participation from RA Capital Management, Invus, Acuta Capital Partners, an investment fund associated with SVB Leerink Prior Locanabio investors ARCH Venture Partners, Temasek, Lightstone Ventures, UCB Ventures and GV, also participated in the financing round.

"We are pleased that a team of highly sophisticated investors led by Vida Ventures has joined in this financing round, further validating our progress in research and the significant potential of our unique RNA-targeting platform, Burns said in a statement.

With the Series B, Rajul Jain, director of Vida Ventures, joined Locanabio's board of directors. Before Vida Ventures, Jain served on the executive team and headed development for Kite Pharma and was previously global development lead for Amgen.

The unique approach in RNA targeting using gene therapy to deliver RNA binding proteins developed by Locanabio represents the next frontier of genetic medicine with the ability to target the root cause of a range of genetic diseases, Jain said in a statement. They have built a strong management team to execute this bold vision and we are proud to support them.

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Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies - BioSpace