Archive for the ‘Gene Therapy Research’ Category

EXTON, Pa., Jan. 10, 2022 /PRNewswire/ -- Castle Creek Biosciences, Inc., a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for rare genetic diseases, today announced it has acquired Novavita Thera, Inc., a preclinical gene therapy company focused on rare liver and metabolic diseases. The acquisition expands Castle Creek's technology platform by adding in vivo capabilities to its existing ex vivo approach, and broadens Castle Creek's development pipeline beyond skin and connective tissue disorders to rare liver diseases.

"This acquisition is a significant inflection point for Castle Creek and positions us to expand our research and development efforts using a versatile, dual technology platform that will accelerate the discovery of disease-modifying and potentially curative therapies for people living with rare diseases," said Matthew Gantz, president and chief executive officer of Castle Creek Biosciences. "The ability to leverage both ex vivo and in vivo based approaches is a distinct advantage that few cell and gene therapy companies can offer. We are now in position to pursue new indications for devastating rare diseases, while also advancing our ongoing pivotal clinical trial in recessive dystrophic epidermolysis bullosa (RDEB)."

With the acquisition of Novavita Thera, formerly aCytotheryx, Inc., company, Castle Creek will initially develop a gene therapy for hereditary tyrosinemia type 1 (HT1),a rare inborn error of metabolism caused by a lack of the enzyme fumarylacetoacetate hydrolase (FAH) which leads to accumulation of tyrosine and its metabolites in the liver. HT1 affects approximately 1:100,000 live births and leads to cirrhosis, liver failure, hepatocellular carcinoma, and is ultimately fatal if untreated. Liver transplantation is currently the only curative treatment available for HT1.

Castle Creek will advance the development of LV-FAH, a potential therapy based on a lentiviral vector containing a functional copy of the human FAH gene that is administered directly to the patient through the portal vein. The therapy is designed to transduce hepatocytes and deliver the FAH enzyme that is deficient in these cells.Castle Creek plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for LV-FAH in HT1. Castle Creek also continues to progress several additional candidates targeting other rare liver and metabolic diseases and skin and connective tissue disorders.

In connection with the acquisition, Joseph Lillegard, MD, PhD, has joined Castle Creek as chief scientific officer. Dr. Lillegard is a board-certified pediatric and adult general, thoracic and fetal surgeon at the Children's Hospital of Minnesota, and led the cell and gene therapy research lab at Mayo Clinic that discovered LV-FAH. Robert A. Kaiser, PhD, DABT, has also joined the company as vice president of preclinical development. Dr. Kaiser is a board-certified toxicologist with over a decade of experience designing, conducting, and reporting preclinical and IND-enabling studies. Dr. Lillegard and Dr. Kaiser will be the company leads for Castle Creek's recently announced research collaboration with Mayo Clinic to advance discovery and development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome.

"It is an exciting time to join Castle Creek, a company that has already established an impressive research and development program in cell and gene therapies with proven clinical development and in-house manufacturing capabilities," said Dr. Lillegard. "I look forward to collaborating with the company's dedicated team on development of novel gene therapies. We believe our work to evaluate the safety of in vivo lentiviral vector administration in HT1 has the potential to be a precedent setting approach that can be applied to a range of new therapeutic areas for underserved patient populations."

About Castle Creek Biosciences, Inc.

Castle Creek Biosciences, Inc. is a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for patients living with rare genetic diseases. Castle Creek's most advanced product candidate, dabocemagene autoficel (FCX-007, D-Fi), an ex vivo, autologous gene therapy, is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also evaluating FCX-013, an ex vivo, autologous gene therapy, in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma.In addition, LV-FAH, an in vivo, investigational gene therapy candidate, is being assessed in preclinical studies for the treatment of hereditary tyrosinemia type 1 (HT1).Castle Creek is pursuing discovery and development of early-stage novel product candidates utilizing its dual platform of ex vivo and in vivo technologies to expand its robust pipeline. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania. Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC. For more information, visit https://castlecreekbio.com/or follow Castle Creek on Twitter @CastleCreekBio.

About Paragon Biosciences, LLC

Paragon is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, adaptive biology and advanced biotechnology. The company's current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

Media Contacts

Adam DaleyBerry & Company Public Relations212.253.8881[emailprotected]

Karen CaseyCastle Creek Biosciences302.750.4675[emailprotected]

SOURCE Castle Creek Biosciences, Inc.

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Castle Creek Biosciences Acquires Novavita Thera to Expand Innovative Cell and Gene Therapy Platform - PRNewswire

Risky biotechnology start-ups that soared in recent years can't catch a break in 2022. Beam Therapeutics (NASDAQ:BEAM) recently signed a major research deal with Pfizer (NYSE:PFE) that could be worth up to $1.35 billion, and hardly anyone seemed to notice.

This was clearly great news for Beam Therapeutics, but a stock market scorned for clinical-stage biotech stocks didn't respond the way anyone familiar with the company would have expected. Instead of surging higher in response to the Pfizer deal, Beam Therapeutics stock actually fell nearly 2% on the day of the announcement.

Nearly all biotech stocks are in the doghouse lately and it looks like the market may have missed something here. Let's look closer to see if Beam Therapeutics is a smart buy at the moment.

Image source: Getty Images.

Over the past few years, Pfizer has watched its peers experiment with CRISPR-based gene editing techniques without making any significant investments. Beam Therapeutics' base-editing technology, though, really got the big pharma company's attention.

Instead of removing and replacing entire sections of genetic material like Intellia, andCRISPR Therapeutics (NASDAQ:CRSP), Beam Therapeutics is pioneering a more precise method called base editing. This involves altering just one letter of genetic material at a time, which is a lot more useful than it might seem. Around half of all known genetic variations associated with diseases are caused by single-point mutations.

Pfizer will give Beam Therapeutics a $300 million payment up front to discover new drug candidates aimed at three undisclosed targets that won't compete with Beam's existing programs. Beam's eligible for up to $1.05 billion in milestone payments if all three go on to become a commercial success.

Beam Therapeutics is eligible to receive royalties at an undisclosed percentage of global sales for each future program. Beam Therapeutics even has an option to co-develop and co-commercialize one of the future candidates for a larger cut of sales.

Beam Therapeutics finished September with $934 million in cash after operations burned through $329 million during the first nine months of the year. Pfizer's cash injection should raise the company's cash balance high enough to get through 2023 before it needs to tap investors for more.

There's no telling whether Pfizer will decide to license a candidate from Beam Therapeutics. If Pfizer drags its feet, the gene-editing start-up has some preclinical-stage programs of its own that might have a chance to impress investors before it's time to raise capital again.

The most advanced candidate in Beam's pipeline at the moment, BEAM-101 is an experimental gene therapy for the treatment of sickle cell disease. The company doesn't expect to begin enrolling patients into the first clinical trial with BEAM-101 until the second half of 2022.

With a recent market cap of $4.5 billion, there's already a lot of success for Beam's pipeline priced into the stock. Unfortunately, the road ahead could be a lot more challenging than investors are anticipating. Last year, Vertex Pharmaceuticals (NASDAQ:VRTX) and collaboration partner CRISPR Therapeutics reported highly encouraging results from a clinical trial with CTX001 that started way back in 2018.

CTX001 is an experimental gene therapy for sickle cell disease that's similar to BEAM-101 in that it encourages the production of fetal hemoglobin. If early interim data that Beam Therapeutics posts a couple of years from now doesn't appear competitive with CTX001, the stock could take a tumble. While this is a top gene-editing stock to watch, it's still a little too risky to buy right now.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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Is Beam Therapeutics a Good Stock to Buy Now? - The Motley Fool

Amicus Therapeutics is moving on from an early-phase Batten disease program after follow-up data showed the therapy didn't stop the fatal nervous system disease from progressing long-term.

The company disclosed the pipeline trim in a preliminary revenue guidance announcement ahead of its Wednesday presentation at the J.P. Morgan Healthcare Conference.

Amicus was advancing a gene therapy program for CLN6 Batten disease, a type of the rare genetic disorder that causes development regression and typically begins in childhood. Back in 2019, Amicus shared data on AAV-CLN6 that showed the gene therapy stabilized children's motor and language functions.

But the CLN6 program will now be discontinued after the company got a look at long-term extension data from a phase 1/2 trial.

RELATED:Amicus shares early look at Batten disease gene therapy

The company found the disease stabilization that had occurred during the earlier portion of the trial was not sustained at the two-year mark. Amicus plans to review the data with the CLN6 Batten disease community to support continued research efforts to find better treatments and cures which are so desperately and urgently needed, according to the guidance announcement.

During its last earnings report in November 2021, Amicus had said it would be ramping up manufacturing activities and regulatory discussions for the program.

Meanwhile, the company will advance its CLN3 Batten disease program, which is currently in a phase 1/2 trial. A readout from the trial and additional preclinical data are expected in 2022, Amicus said. Once the data are released, the company can begin work on a pivotal trial.

Continued here:
JPM 2022: Amicus axes gene therapy program for type of Batten disease, advances another - FierceBiotech

I said, I am an RNA scientist. I can do anything with RNA, Dr. Karik recalled telling Dr. Weissman. He asked her: Could you make an H.I.V. vaccine?

Oh yeah, oh yeah, I can do it, Dr. Karik said.

Up to that point, commercial vaccines had carried modified viruses or pieces of them into the body to train the immune system to attack invading microbes. An mRNA vaccine would instead carry instructions encoded in mRNA that would allow the bodys cells to pump out their own viral proteins. This approach, Dr. Weissman thought, would better mimic a real infection and prompt a more robust immune response than traditional vaccines did.

It was a fringe idea that few scientists thought would work. A molecule as fragile as mRNA seemed an unlikely vaccine candidate. Grant reviewers were not impressed, either. His lab had to run on seed money that the university gives new faculty members to get started.

By that time, it was easy to synthesize mRNA in the lab to encode any protein. Drs. Weissman and Karik inserted mRNA molecules into human cells growing in petri dishes and, as expected, the mRNA instructed the cells to make specific proteins. But when they injected mRNA into mice, the animals got sick.

Their fur got ruffled, they hunched up, they stopped eating, they stopped running, Dr. Weissman said. Nobody knew why.

For seven years, the pair studied the workings of mRNA. Countless experiments failed. They wandered down one blind alley after another. Their problem was that the immune system sees mRNA as a piece of an invading pathogen and attacks it, making the animals sick while destroying the mRNA.

Eventually, they solved the mystery. The researchers discovered that cells protect their own mRNA with a specific chemical modification. So the scientists tried making the same change to mRNA made in the lab before injecting it into cells. It worked: The mRNA was taken up by cells without provoking an immune response.

More:
How The mRNA Vaccines Were Made: Halting Progress and Happy Accidents - The New York Times

Leptin is a hormone that comes from fat cells. It helps control food intake by sending signals about hunger to the hypothalamus in the brain. This process regulates appetite.

Leptin regulates energy levels by maintaining a balance between hunger and appetite. The hormone triggers the body to respond by eating more when energy levels are low and eating less when energy levels are stable or high.

People who have high levels of body fat have high circulating levels of leptin.

Research shows that having elevated leptin levels can lead to leptin resistance, making weight loss difficult.

This article looks at what the leptin hormone is, what the leptin diet involves, and the advantages and disadvantages of following the leptin diet.

Scientists discovered leptin, a protein that functions as a hormone, in 1994.

Leptin is one of the main hormones responsible for maintaining body weight. Leptin helps people balance how much food they consume by regulating hunger levels. The hormone also controls how much energy a person uses throughout each day.

Leptin comes from fat cells within the body. It enters the blood supply and travels up to the brain. The hormone must cross the blood-brain barrier, a membrane that protects the brain from harmful toxins, to get to the hypothalamus. The hypothalamus is the area in the middle of the brain that controls hormone regulation, among other important functions.

At the hypothalamus, leptin can function by signaling that the body does not need any more food. This response causes the person to feel full. If leptin levels are low, or leptin does not reach the hypothalamus, a person will continue to feel hungry.

Leptin regulates body weight and is an important marker for energy storage. This means if the body has excess energy stored as fat, leptin signals the hypothalamus to reduce appetite and burn excess body fat for fuel. This response helps a person maintain a moderate body weight.

However, when a person has high amounts of body fat, they can develop a resistance to leptin, which leads to abnormally high leptin levels.

Having leptin levels that are too low is less common. Low leptin levels can occur in severe childhood obesity and delayed puberty.

When leptin levels are below average, the brain thinks no body fat is present. This reduced level can cause symptoms of uncontrollable hunger, resulting in excessive food intake. Leptin injections are a way of reducing this problem.

After scientists discovered the hormone in 1994, Byron J. Richards created a diet named after it: the leptin diet.

The goal of the leptin diet is to return leptin levels to normal and create balance within the body. The leptin diet has five main principles:

The leptin diet permits most types of food, but guidelines suggest avoiding chemical additives and processed sugars and sticking to fresh and organic produce.

The leptin diet encourages other lifestyle changes, such as getting plenty of sleep and participating in regular physical activity.

A 2021 study suggests that diets high in fat, carbohydrates, fructose, and sucrose and low in protein are drivers of leptin resistance. The researchers concluded that leptin resistance might be reversible by reducing calories.

However, this research has some limitations, such as small sample sizes, so further evidence is required to verify these claims.

The leptin diet includes limiting snacking and shortening your daily eating window. If a person reduces how much they snack, this could create a calorie deficit necessary for weight loss.

A leptin diet is a sensible approach to weight loss for some people, as the diet promotes eating healthily without harsh restrictions but encourages a routine.

However, at present, no studies are investigating the effects of the leptin diet on weight loss and leptin levels.

It is important to remember that all bodies are different, and a diet that meets the nutritional demands of one person will not always work for someone else.

For example, limiting the number of meals to three per day and cutting out snacking may be effective for a person with a low activity level. However, it is unlikely to meet the energy demands of a person who leads an active lifestyle, exercises intensely, or has a physically demanding job.

Many factors can impact energy needs, including age, pregnancy, breastfeeding, and certain medical conditions.

A person should consider consulting a healthcare professional like a registered dietitian if they are interested in improving health through dietary changes.

Increasing research suggests that obesity causes people to develop leptin resistance.

When someone carries an excessive amount of body fat, they will have too much leptin circulating in the blood. This excess results in that person becoming leptin-resistant. This resistance means their brain stops responding to the leptin signals traveling up to it. It also means their body continues to produce leptin, contributing to elevated leptin and leptin resistance.

Research suggests that weight loss and energy-restricted diets may help reverse leptin resistance.

Leptin is the hormone that controls appetite. Leptin informs the brain when a person has eaten enough, reducing appetite, and produces hunger signals when a person requires energy.

As with any weight loss plan, a person should approach the leptin diet with caution. The diet may be effective for some people, but it may not meet the nutritional demands of every person. Check with a doctor before starting any significant weight loss diet.

Link:
Leptin: Benefits and risks of the leptin diet - Medical News Today

CAR-T cell therapy has been a boon for treating blood cancers. Since the technology was first brought to the clinic, CAR-T has offered patients months or years of life after they had exhausted all other treatment options and would have died within weeks.

Its been incredible, said Marcela Maus, an immunologist and cell therapist at Mass General Cancer Center. Weve seen patients who had multiple lines of therapies and progressed after all of those, [then] get CAR-T and go into long-term remission.

But CAR-T does have hefty limitations, and scientists like Maus are researching ways to overcome some of its major shortcomings. These issues have prevented CAR-T from being used safely and effectively outside of leukemia and myeloma, and even patients who have responded spectacularly to CAR-T usually see their cancers return. The therapies are also still incredibly costly and carry risks, including a reaction known as a cytokine storm that can be life-threatening.

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Potential solutions to these problems are still in the early stages, but scientists are beginning to get a vision of what the future of CAR-T cell therapy might look like. It could involve synthetic biology to engineer a more advanced cell, or engineering other parts of the T cell to make it work better in the challenging environment around a tumor.

The field is growing tremendously, Maus said. Different people are working on different issues then, ideally, the data kind of decides whats going to be the next big thing.

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Heres a look at what experts see as some of the most promising approaches.

Current CAR-T cells use their CAR, or chimeric antigen receptor, to identify and kill cancer cells. These are synthetic proteins that bind to a specific target, like a protein on a cell surface membrane, and then activate the T cell to kill any cell carrying this target.

Armed with a CAR, T cells become pros at killing cancer cells that have their target, but theyll also kill normal cells that happen to carry the protein, too. Once a CAR-T cell is in the body, there isnt much a clinician can do to rein it in if it starts causing a lot of toxicity.

Once we let the CAR out, theyre like teenage kids, Maus said. You can maybe watch, but you cant really control them. So, theres some desire to be able to turn them on or off at will.

So, researchers are also trying to create CAR-T cells that they can manually activate or deactivate. As a group, these are known as controllable CARs, and most work by engineering an additional genetic circuit in the CAR-T cell. In theory, clinicians should be able to activate a switch on the genetic circuit that induces the CAR-T cell to activate their CAR and express it on the T cells surface membrane, thereby activating the receptor. Then, after a while, the T cell will disarm.

The goal is really getting our hands back on the steering wheel for a bit, Maus said.

There are several ways that synthetic biologists are doing this. In one example, researchers engineered a CAR with a protein switch that activates the receptor in the presence of blue light. In another example, researchers added a gene to CAR-T cells that force it to create its CAR and express it on the cell surface, thereby activating it, only in the presence of ultrasound radiation.

That way, it can be focused into a specific location, said Peter Yingxiao Wang, a synthetic biologist at University of California, San Diego, who works on controllable CARs. When the light or ultrasound is on the tumor locally, they can activate the CAR gene to trigger killing. Anywhere else, the CAR T-cells will be benign.

The idea is that the clinician can focus the light or ultrasound onto the tumor to get CAR-T cells to begin killing there. Once that signal is turned off, the CARs should disarm or slowly degrade and deactivate the CAR-T cells killing function. This way, even if the CAR does kill healthy tissue, the damage will theoretically be limited to the area around the tumor.

But this is an infant field right now, Wang added. A lot of these studies are just proof of concepts to show that theyre technically achievable. If you want to move to clinical trials, all of the components must be optimized.

Scientists also must show that theyre truly safe in humans, and that keeping the damage to a smaller surface area will be enough to outweigh the risks in treating tumors located near vital organs like the heart.

Other researchers are working on developing new CARs that can function like a biomolecular computer, able to make simple logical decisions to target cancer cells. Conventional CARs can cause dangerous toxicity because they only use one protein to identify cancer cells, and it may be impossible to discover the perfect target that exists only on cancer cells and not at all on healthy cells.

You can never uniquely define cancer or any other healthy tissue just by one marker, explained Wilson Wong, a synthetic biologist at Boston University. It just doesnt work. Its like trying to find a person and saying, he has black hair. Its like, oh, my God, youll never find him.

But it might be possible to distinguish cancer cells from healthy ones by looking at multiple proteins on a single cell. So, researchers like Wong have begun building more advanced CAR T-cells that use genetic circuits that only activate a CAR under more complex conditions, like the presence of several specific proteins that arent often seen in combination on healthy cells.

In this sense, the CAR is making a logical decision like basic Boolean computing, and synthetic biologists call this technique logic-gating.

Theres a lot of cool genetic circuits you can build, said Yvonne Chen, a synthetic biologist at UCLA. One can think of conditional systems to obliterate cancer cells. One can build OR-gates, AND-gates, and NOT-gates, and layer them on top of one another.

Although, Chen added, a drawback of logic-gating is that by increasing the complexity of the system, you might also be increasing the chance something goes wrong. Its important not to overcomplicate the design. Sophisticated circuits are exciting, but sometimes the solution itself causes problems. For example, for an AND-gate, you also make it easier for the tumor to escape. If the tumor loses either target A or B, it escapes from therapy, she said.

Another issue with conventional CAR-T therapy is that after a while, T cells can simply stop working. Solid tumors, like lung or pancreatic cancer, often have strategies to defend themselves from immune system attacks, including those from CAR-T cells. That makes it harder for CAR-T cells to treat solid tumors and can provide an opening for the tumor to return or progress.

So, researchers like Chen are working on armoring the CAR T-cell against the hostile signals in the microenvironment around a solid tumor. One of these signals is called TGF-beta, a protein which can help shut down T cell activity and help cancer cells avoid death and detection from the immune system. Chen was able to create a CAR cell that is not only resistant to TGF-beta, but can actually subvert the signal and become more deadly when it encounters TGF-beta.

Instead of being dysfunctional, they become activated, Chen said. That actually converts a tumor defense mechanism into a stimulatory signal for our T cells and tells them, youre in an environment where youre likely to encounter a tumor cell. Get ready.

Other scientists are working to keep CAR-T cells which can lose power over time functional for longer. Even with a good antigen, the T cells rapidly lose function, said Shivani Srivastava, an immunologist at the Fred Hutchinson Cancer Research Center who works on this problem. If you trigger a T cell or CAR over and over again, that causes the cell to become exhausted rather than turning into a memory cell or something else.

In one case, Stanford immunologist Crystal Mackall engineered a CAR-T cell that takes breaks before returning to work. She did this by creating a transient CAR that can be turned on or off. It can enhance [the T cells] function and limit how exhausted they are by giving them periodic rest, Srivastava said. Thats a really interesting strategy in principle.

But most of the tactics that scientists have tried so far in the realm of armored CAR-T cells havent worked in the long term, Srivastava said. You need a strategy that can help the CAR T-cells persist long enough to eradicate the cancer and prevent its return, which might be a lifelong project for the immune system.

Well have to find the right combination that will be durable, she said. Often we can find strategies that enhance function for only a short period of time.

Some future approaches might see T cells abandoned altogether. Scientists are slapping synthetic receptors on new or different cell types, such as natural killer cells. One company, called CoImmune, is putting CARs on a synthetic cell called a CIK cell, or cytokine-induced killer cell.

This is a novel cell type. They dont occur in nature, explained Charles Nicolette, the biotechs chief executive.

Theyre made by taking white blood cells and growing them while exposing them to certain immune molecules called cytokines. The advantage of creating new cell types is that biologists can combine certain useful traits from other immune cells, Nicolette said. For example, CIK cells could have the NK cells natural ability to distinguish normal cells from malignant ones and the CAR T-cells enhanced ability to kill.

One day, UCLAs Chen hopes to take this concept even further. To her, the ideal cancer-killing cell would not be derived from anything biological, but a completely artificial cell.

Instead of taking a cell from a patient, but rather build a completely defined, minimal cell that can do what we want and nothing else. It cannot evolve. Cannot mutate. Then, self-destruct when you dont want it there, she said. But, she added, creating synthetic cells like that would be unimaginably challenging, and it might not be possible to create a cell thats both persistent but also unchangeable.

Still, a scientist can dream.

Read more:
STAT's guide to the next generation of CAR-T therapies - STAT

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The research covers the current Cancer Gene Therapy market size and its growth rates based on records with company outline of Key players/manufacturers: GlaxoSmithKline plc, Adaptimmune Therapeutics plc, Merck & Co., Inc., bluebird bio, Inc., Shanghai Sunway Biotech Co., Ltd, Celgene, SIBIONO, Anchiano Therapeutic, Achieve Life Sciences, Inc., and Synergene Active Ingredients Pvt. Ltd.

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Cancer Gene Therapy Market 2021 Industry Outlook, Comprehensive Insights, Growth and Forecast 2031 | Celgene, SIBIONO, Anchiano Therapeutic, Achieve...

Just like at last year'sJ.P. Morgan Healthcare Conference,M&A is so 2019.

In the third winter of COVID-19, the drug development world hasnt executed on the type of megamergers we saw in 2019, such as Bristol Myers Squibbs $74 billion Celgene deal,which dropped in the lead-up to the healthcare industry's largest gathering.

Of course, we don't know whats happening in private Zoom rooms at the moment, or who managed to make some company-defining contacts at the second virtual edition of the conference.

Galapagos, with $5.6 billion to spend and a thinned late-stage pipeline, made it known Thursday morning that there are, in fact, private Zoom meetings to hash out potential acquisitions. The biopharmas chief business officer wasnt shy to say Galapagos is speaking with a host of companies as the company works on a rebound for 2022.

RELATED:2022 forecast: Biopharma M&A lags in 2021. Will drugmakers still look for bolt-on deals or large transactions?

But what was made public is that biopharmas stuck to licensing and research partnerships this week at JPM, extending a trend that has been going on for months. Backloaded, billion-dollar biobucks pacts are increasingly standard.

This year, I think, is a reflection of last year, and its much more licensing, much more tie-up, said Stuart Henderson, global life sciences industry leader at Accenture, in an interview with Fierce Biotech.

There are a few key factors driving the focus on licensing and collaborations, according to Henderson.

Premiums on acquisitions have increased, thus limiting affordability; venture capital funding has skyrocketed three times in the past five years, reducing the need for pharmas money; biotechs are going all the way to market on their own with tighter labels and more defined populations, shrinking the need for pharmas help in selling drugs; and the economic value of Big Pharma buying a late-stage biotech has declined, he said.

The virtual conference started with multiple licensing and research handshakes revealed Monday morning. Bayer kicked off the week with a $1 billionbiobucks beton a gene therapy deal with Mammoth, using the CRISPR science out of the lab of Jennifer Doudna, Ph.D.

RELATED:JPM22, Day 3: In short order, Perrigo part of a new landscape; Legend, J&J prep for cilta-cel launch

Next up was Novartis,ponying up$163 million to opt in on a COVID-19 antiviral after its collaborator Molecular Partners showed a 78% risk reduction in early-stage patients. Then, Pfizerdoled out$300 million upfront for Beam Therapeutics base editing technique. The COVID-19 vaccine maker madeanother two dealsMonday morning, including a financials-free agreement with Acuitas Therapeutics for its lipid nanoparticle delivery system for use in mRNA vaccines and therapies. Add to that a $100 million biobucks pact with Codex DNA.

This was allbefore 7 a.m. ET on Monday. Pfizers pandemic rival, Moderna, made some waves with its owndevelopment dealat $45 million upfront for Carisma Therapeutics in vivo engineered chimeric antigen receptor monocyte therapeutics in cancer. BioNTech followed with a $750 million milestone paymentspactwith Crescendo Biologics; Bristol Myers Squibb offered awhopping $3 billionin biobucks for cell therapies from Century Therapeutics; and Acadia was stoked tosigna $60 million upfront tie-up with Stoke Therapeutics.

Thats at least nine collaboration and licensing deals in the first day. You get the pattern. It was medtech that brought the M&A: Medtronicoffered$925 million for cardiac mapper Affera, and Exact Sciences willspend $190 millionon testing lab PreventionGenetics.

Henderson said some of the biotechs attracting licensing interest are those with platform technologies and digital biology companies that are attempting to cut the billion-dollar costs of drug discovery and development.

RELATED:JPM22, Day 4: Vertex aims to be cystic fibrosis leader into the 2030s; Gritstone hopes for new cancer biomarker; Galapagos' rebound year

The licensing announcements have slowed since those first days. But could that meanarms will be linked in coming weeks, as final negotiations are hashed out and due diligence is conducted? We hope so.

We know pharma is looking. Plenty of companiesPfizer, Moderna, BioNTech, Novartis and many morehave cash burning a hole in their pockets and pipelines to fill.

Read more:
JPM 2022: Licensing and research tie-ups trump M&A at conference in 'reflection of last year' - FierceBiotech

The evolution of advanced therapy medicinal products, or ATMPs, is bringing real world results, write James Fry, partner, and Isabel Teare, senior legal adviser from leading law firm Mills & Reeve.

The number of cell and gene therapy products now on the market around the world is near the 100 mark, according to recent research by McKinsey & Company, with many more in development.

These ground-breaking therapeutics can have a profound impact on previously intractable conditions. Products like Novartiss gene therapy Zolgensma, for example, are costly but have the potential to act as a single-dose cure for young patients with spinal muscular atrophy.

London-based Orchard Therapeutics has had some important wins with its ex vivo autologous gene therapy approach, as it seeks to address multiple therapeutic areas with profound unmet need.

The UKs powerful research base and established life sciences sector means that it is well placed to be part of this story. The Cell and Gene Therapy Catapult reports that, in 2020, the UK was host to around 12 per cent of ongoing ATMP global clinical trials some 154 separate studies. A striking statistic.

As more products are rolled out to patients, the journey for others both in overcoming technical obstacles and gaining public acceptance improves. But in the global race to bring these sophisticated products through from concept to clinic, some specific challenges stand in the way.

One of these is the need for a different style of regulatory engagement. The traditional methods used by regulators in dealing with small molecule pharmaceuticals show major limitations when applied to ATMPs.

Regulators have many years of in-depth experience with small molecule medicines. This can mean that regulatory guidance focuses on this section of the market and tends to be prescriptive in nature.

In contrast, regulation of large molecule, cell and gene therapies should, say those close to the industry, be based more on a philosophy rather than prescriptive rules, and should allow a greater degree of flexibility.

To remain at the forefront of this exciting sector, rapid technological progress needs to be matched by a surefooted regulatory response. With many ATMP projects created in the hands of smaller spin-outs or growing mid-sized companies there is no spare cash or capacity for mistakes.

Developers and regulators need to work together to make sure that safe and effective therapies can reach the patients that so badly need them.

A collaborative approach

Developers of innovative therapies can gain real benefits from early and regular engagement with regulatory agencies. The aim here is to smooth the product development path and, at the same time, avoid wasted effort and expense.

With development costs high, collaboration is a must to drive down complexity and uncertainty. The pressures of COVID-19 pandemic may have helped in strengthening the collaborative approach between regulators and developers, and many hope to see these changes embedded in day-to-day practice.

Think holistically

Regulatory strategy needs to form part of the overall development picture. For example, the shelf-life of ATMPs, and also their starting materials, can be very short.

Building an understanding of the practical issues into the supply chain is essential. Issues around cross-border transfers with possible delays for customs checks can seriously undermine the business model for this generation of products.

Likewise, point of care manufacturing may be a necessary element of treatment something regulators may allow for if they appreciate its importance.

New ways to shape clinical trials

Designing clinical trials of ATMPs to make sure that they are not excessively cumbersome or open-ended requires early planning and engagement.

Regulators are working on tools that can help innovators in this area, such as novel trial structures like basket or umbrella trials.

These can be deployed to evaluate multiple hypotheses with the overall goal of improving the efficiency of trial evaluation.

As clinical development progresses it may become necessary to adapt the trial, and regulators need to recognise this. Long term follow-up may also be necessary.

Regulators learning from each other

With advanced therapies setting new challenges, developers can take heart from seeing regulators learning from each other and sharing lessons internationally.

Many products in this area address rare diseases, so a coordinated approach makes sense in serving a group of patients spread around the world.

Likewise, regulators can benefit from looking to novel examples elsewhere.The Japanese authorities offer a special conditional approval that looks at safety and predicted efficacy, before allowing products a provisional authorisation for the market. This enables treatment availability to patients for a number of years during which evidence to establish efficacy is gathered.

An unexpected benefit

The COVID-19 pandemic, while stretching healthcare provision to its utmost, has had some positive impacts. Widespread recognition of the importance of life science innovation is one.

But accelerating new approaches and ways of working with and between regulators is another less obvious benefit. We have looked at how this can strengthen and support cell and gene therapy development and are optimistic that these changes are here to stay.

And the UK governments recent Life Sciences Vision shows a real commitment to pushing through in many of these areas.

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Cell and gene therapies: How to encourage and promote the move to market - Business Weekly

Researchers atUniversity Health Network (UHN) and the University of Toronto have received $24 million to advancetechnology to repair and rebuild organs outside the bodyfor patients in need.

The project, led byShaf Keshavjee, is one of only seven across Canada selected to receive funding in the Government of CanadaNew Frontiers in Research Fund(NFRF) Transformation competition, following an international consultation.

"The Ex Vivo Lung Perfusion (EVLP) system we developed here in Toronto has revolutionized lung transplantation in the past decade. Now, it's been translated around the world to increase lung transplant access and it's being extended to other organs," says Keshavjee, a professor and vice-chair for innovation in thedepartment of surgeryin U of Ts Temerty Faculty of Medicinewho is surgeon-in-chief at UHN and a senior scientist atToronto General Hospital Research Institute.

"With this transformative grant, we now have the opportunity to take ex vivo technology to the next level, where we can repair and rebuild organs for transplant."

Atul Humar, director of the AjmeraTransplant Centre(photo byTim Fraser)

Over 4,500 people in Canada are currently waiting for an organ transplant, and more than 270 die each year as the need for transplant greatly exceeds availability.

Ex vivo perfusion systems use specialized machines to maintain, evaluate and treat organs before transplant. They have a huge impact on increasing the number of organs that can be considered for transplant.

TheToronto Lung Transplant Program,led by Keshavjee, has used this technology to double the number of lung transplants performed and lives saved at UHN.

"The New Frontiers grant will allow us to advance applications for lungs and further develop ex vivo systems for other organs, such as liver, kidney, heart and pancreas," says Atul Humar, a co-principal investigator on the project, professor in thedepartment of medicineat U of T and director of theAjmera Transplant Centre at UHN.

Brad Wouters, UHN's executive vice president, science and research, notes that this major grant will enable multidisciplinary teams to develop new, cutting-edge approaches to extend the time that donated organs can be used, and also enable treatment and repair of unsuitable organs to allow treatment of more patients.

It will also help the teams refine and improve equitable organ allocation guidelines for all patients, he adds.

The advancements that this team has made and their continued success is made possible by support from provincial and federal governments, industry partners, external charitable agencies, generous philanthropy from the UHN Foundation and our incredible patient partners, says Wouters, who is also a professor in thedepartment of radiation oncologyat U of T. This award recognizes the tireless efforts of the team, and this support, which have been key to achieving global impact.

The New Frontiers Research Fund was designed to support large-scale, Canadian-led interdisciplinary research projects with the potential to realize real and lasting change.

The fund falls under the strategic direction of theCanada Research Coordinating Committeeand is administered by the Tri-Agency Institutional Programs Secretariat on behalf of Canada's three research granting agencies: theSocial Sciences and Humanities Research Council, theCanadian Institutes of Health Researchand theNatural Sciences and Engineering Research Council.

Over the course of this project, the team of over 20 researchers at U of T, UHN, national and international partner sites will develop sophisticated ex vivo platforms to:

Longer ex vivo preservation prior to transplant will enable many world-first therapeutic applications that will, ultimately, create more organs for clinical transplant.

One example is to use gene therapy to make an organ more like the recipient's cells and help to address the current hurdle of organ rejection by the immune system. Researchers at UHN are also working on changing an organ's blood type so the sickest people can get access to the next available organ, instead of waiting for one that exactly matches their blood a delay that currently can take several months before a match is found.

Another transformative goal is to use medicines and light therapies in the ex vivo circuit to eliminate viral or bacterial infections that previously prevented an organ to be considered for transplant.

"This grant gives us a unique opportunity to extend personalized medicine to every organ group," saysMarcelo Cypel, a professor in the department of surgery at U of T and surgical director of the Ajmera Transplant Centre, who is also a co-principal investigator on the project.

"Not only will it enable longer preservation, this research will let us treat and improve organs. It has the potential to change the paradigm in the field of transplantation."

The change will include several advanced applications, such as the engineering of new organs using stem cells with the goal to make organs available for all in need. Significant progress has already been made in generating new kidneys, lungs and tracheae (windpipe), and their applications will be tested further during the six-year project term.

With the involvement of a multidisciplinary team housed in a world-class centre at UHN, the project will bring personalized medicine to transplant, and go beyond solid organs.

Siba Haykal, plastic and reconstructive surgeon and project co-principal investigator, will lead research involving vascularized composite allotransplantation the transplant of limbs, face, trachea and composite tissues, such as skin and muscles.

"These are very delicate tissues that can't survive outside the body for very long and are very susceptible to rejection," she explains, adding that the current treatment involves high doses of life-long anti-rejection medication for transplant recipients.

Haykal and the team want to develop a system to preserve limbs and tissues out of the body without blood flow for longer periods. This will enable the application of new cell therapies to adapt these tissues to the recipient prior to surgery.

"Whether they have been disfigured by burns or from trauma or cancer, if they've had an amputation and need prosthetic limbs or if they require a new airway, transplantation provides hope for these patients who currently don't have many options," says Haykal, who is an assistant professor in the department of surgery at U of T.

"If we can use techniques that reduce the amount of anti-rejection medication and maybe one day get to a stage where they don't need it anymore, that would have a huge impact on the patient's quality of life."

Humar adds, "I have seen so many people who have literally been at death's door and have been completely turned around by transplant and live a full and healthy life. If we can offer that to more patients, then for me that would be an incredible achievement.

"This funding will also help us disseminate our knowledge, and facilitate other hospitals across Canada and around the world build upon what we're doing at UHN."

This story wasoriginally postedon the University Health Network website.

Continue reading here:
UHN and U of T receive $24-million federal grant for transplant research - News@UofT

DUBLIN--(BUSINESS WIRE)--The "Global Cell and Gene Therapy Manufacturing Services Market 2021-2027" report has been added to ResearchAndMarkets.com's offering.

The global cell and gene therapy manufacturing services market is projected to grow at a potential CAGR during the forecast period (2021-2027).

The key aspect that drives the growth of the market includes the rising incidence of cancer coupled with the increasing R&D by pharmaceutical companies and partnerships & agreements between pharmaceutical companies and contract development and manufacturing organizations (CDMOs). Benefits of partnering with a cell or gene therapy CDMOs include scalability, speed to market, access to technical expertise without overhead costs, and cost-efficiency.

Covid-19 has affected economies and industries in several countries due to lockdown, travel bans, and business shutdowns. The global healthcare industry is one of the major industries facing serious disruptions such as breaks in supply chains and disruptions in manufacturing due to lockdown and office shutdowns. However, an increased number of investigational studies based on cell therapies for the treatment of COVID-19 patients have led to a positive impact on the market.

The global cell and gene therapy manufacturing services market is segmented based on therapy, scale, indication, and end-user. Based on therapy, the market is segmented into cell and gene therapy. Among, therapy, the cell therapy segment is expected to grow at a decent rate during the forecast period.

The attributable factor for the growth of the segment is increasing awareness about cell therapy coupled with the development of genomics methods for cell analysis. Based on the scale, the market is segmented into pre-commercial/ R&D scale manufacturing, commercial-scale manufacturing. Among scale, pre-commercial/ R&D scale manufacturing holds a lucrative share in the market during the forecast period owing to the expansion of cell and gene pipelines across the globe.

North America held a major market position in the global cell and gene therapy manufacturing services market in 2020. The presence of key companies involved in the development of cell and gene therapy and growing investments in the field are some of the key factors driving the growth of the regional market. Europe is the second-largest market in 2020 owing to the presence of a strong workforce coupled with strong facilities.

Further, Thermo Fisher Inc., Merck KGaA, and F. Hoffmann-La Roche Ltd., among others are some of the prominent players operating in the market.

Strategic initiatives may support an increase in the market share of the players during the forecast period. The manufacturers are extensively investing in new technologies. Moreover, new launches & developments, partnerships and collaborations, and mergers and acquisitions are some of the growth strategies adopted by the players to sustain in the highly competitive market.

Key Topics Covered:

1. Report Summary

1.1. Research Methods and Tools

1.2. Market Breakdown

2. Market Overview and Insights

2.1. Scope of the Report

2.2. Analyst Insight & Current Market Trends

2.3. Porter's Analysis

3. Competitive Landscape

3.1. Key Company Analysis

3.1.1. Thermo Fisher Scientific Inc.

3.1.1.1. Overview

3.1.1.2. Financial Analysis

3.1.1.3. SWOT Analysis

3.1.2. Merck KGaA

3.1.3. Lonza Group Ltd.

3.2. Key Strategy Analysis

3.3. Impact of COVID-19 on Key Players

4. Market Determinants

4.1. Motivators

4.2. Restraints

4.3. Opportunities

5. Market Segmentation

5.1. Global Cell and Gene Therapy Manufacturing Services Market by Therapy

5.1.1. Cell Therapy

5.1.2. Gene Therapy

5.2. Global Cell and Gene Therapy Manufacturing Services Market by Scale

5.2.1. Pre-commercial/ R&D Scale Manufacturing

5.2.2. Commercial Scale Manufacturing

5.3. Global Cell and Gene Therapy Manufacturing Services Market by Indication

5.3.1. Cancer

5.3.2. CVD

5.3.3. Orthopedic

5.3.4. Infectious Diseases

5.3.5. Others

5.4. Global Cell and Gene Therapy Manufacturing Services Market by End User

5.4.1. Pharmaceutical and Biotechnology Companies

5.4.2. Academic and Research Institutes

6. Regional Analysis

6.1. North America

6.1.1. US

6.1.2. Canada

6.2. Europe

6.2.1. UK

6.2.2. Germany

6.2.3. Italy

6.2.4. Spain

6.2.5. France

6.2.6. Rest of Europe

6.3. Asia-Pacific

6.3.1. China

6.3.2. India

6.3.3. Japan

6.3.4. South Korea

6.3.5. Rest of Asia-Pacific

6.4. Rest of the World

7. Company Profiles

7.1. bluebird bio, Inc.

7.2. Boehringer Ingelheim International GmbH

7.3. Catalent, Inc.

7.4. Cell and Gene Therapy Catapult

7.5. Innovative Cellular Therapeutics Co., Ltd.

7.6. Charles River Laboratories International, Inc.

7.7. F. Hoffmann-La Roche Ltd.

7.8. FUJIFILM Holdings Corp.

7.9. Miltenyi Biotec B.V. & Co. KG

7.10. Nikon CeLL innovation Co., Ltd.

7.11. Novartis AG

7.12. Oxford Biomedica plc

7.13. Samsung Biologics

7.14. Takara Bio Inc.

7.15. WuXi AppTec Co., Ltd.

For more information about this report visit https://www.researchandmarkets.com/r/66e8ge.

Read more here:
Global Cell and Gene Therapy Manufacturing Services Market Research Report 2021-2027 Featuring Prominent Players - Thermo Fisher, Merck KGaA, and F....

JUPITER, Fla. A fun-loving baby with plenty of energy. That's how Linda Trantham describes her daughter Shandra.

"She loved to go to the beach even when she was a toddler and jump in the sea and go to Dubois Park," Linda said.

But one day that changed.

"I first noticed when we went to England on vacation and there was an indoor pool with a wave machine. And the next wave coming along and she like had a funny walk," she added.

A doctor diagnosed Shandra with Friedreich's Ataxia, known as FA, when she was 12 years old. It's an inherited rare disease that affects the nerves and muscles.

WPTV

Shandra herself noticed the change.

"I really couldn't walk in a straight line anymore and I had scoliosis, a sideways curvature of the spine. I had chest pain with exercise," Shandra explained. " So it's estimated that there are about 15-thousand people with it in the entire world. It's like four to five thousand in the U.S."

Shandra graduated from Jupiter High School and went on to the University of Florida. She's now 24 years old and in the Ph.D. program. She's on a path to help people dealing with neurological diseases.

WPTV

She said she's doing gene therapy research in a lab at UF.

"Working on gene therapy in general, kind of contributes to the project for FA, so it's really nice being in the center of all that and getting to see firsthand all of the things that are happening in the development," she said. "My hope is that science will come far enough along that we can just treat people on an individual basis, it would be really cool.

"I'm amazed and in some ways not surprised she's so inquisitive and into science," said Linda.

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Jupiter High School graduate helping in gene therapy research at UF - WPTV.com

The This research service discusses the cell and gene therapy (CGT) market and highlights some key roadblocks in viral vector manufacturing. While many CGT candidates exist in the pipeline, there is a huge capacity deficit that the industry is collaboratively trying to address.

New York, Dec. 21, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Technology Developments in Viral Vector Manufacturing for Cell and Gene Therapies" - https://www.reportlinker.com/p06192548/?utm_source=GNW

Scalability, costs, reproducibility, and overall process efficiency are some of the main pain points at each step of the viral vector manufacturing process.Many industry stakeholders are capitalizing on innovative, sustainable business models and capacity expansion investments to address shortage issues.

Biotechnology companies, such as Merck, Novartis, and Pfizer, and key contract development and manufacturing organizations, such as Thermo Fisher Scientific, Catalent, and FUJIFILM Diosynth Technologies, are investing in new capacities, expanding capacities, and developing innovative technologies to stay ahead in the CGT market. The research covers emerging technologies and trends, challenges, and opportunities across the manufacturing workflow, from upstream (viral vector production) to downstream (viral vector purification). Key developments in upstream processes for viral vector production include advanced transfection agents, novel plasmids, suspension-adapted cell culture, and stable producer cell lines. The research also discusses the general industry shift toward adopting automation, digitization, and advanced analytical processes, including on-line and in-line analytics and robust real-time analytics, to highlight the importance of analytical tools throughout the value chain. Smart technologies, such as automation and digital tools, and the adoption of artificial intelligence and big data support progress in process control and optimization while improving overall efficiencies and safety. The CGT industry works through orchestrated collaborations to develop reference standards and build process analytical technologies (PAT) to optimize manufacturing further. The research presents a birds eye view of key stakeholders and their innovative platforms and a snapshot of the collaborative ecosystem to understand the CGT industrys dynamic and fast-paced nature.Read the full report: https://www.reportlinker.com/p06192548/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Technology Developments in Viral Vector Manufacturing for Cell and Gene Therapies - Yahoo Finance

RESEARCH TRIANGLE PARK The nearly 30 million Americans who suffer from rare diseases have received some good news.

The National Institutes of Health, the U.S. Food and Drug Administration, and a cadre of pharmaceutical companies and non-profit organizations have teamed up to speed the development of new gene therapy treatments. Its good news for North Carolina as well, which is home to close to 50 gene therapy and rare disease-focused businesses that provide jobs for several thousand Tar Heel residents.

Whats called theBespoke Gene Therapy Consortium or BGTC was launched a little less than two months ago. Its part of the NIHs Accelerating Medicines Partnership (AMP), a public/private collaboration to speed drug development across different diseases. Ten global pharmaceutical companies and half as many non-profit patient organizations, as well as 11 NIH institutes, centers and initiatives have signed on.

The project is managed by the Foundation for the National Institutes of Health, whose mission is to promote biomedical discoveries that improve peoples lives.

Currently there are about 7,000 rare diseases in this country, 5,000 of which are due to genetic factors, according to BGTC. A single damaged gene causes nearly 80% of rare genetic illnesses, leaving millions of patients in the U.S. to suffer without much hope of improvement. Currently, only two of these diseases have FDA-approved gene therapy treatments.

BGTC said most rare inherited diseases stem from a specific gene mutation that is already known, which makes gene therapy a promising solution.

A customized or bespoke therapy could correct or replace defective genes with functional ones, according to NIH Director Francis S. Collins, M.D., Ph.D. There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies, he said.

But development is costly, complex and time consuming. And rare disorders by definition affect only a small number of patients. So most pharma companies arent willing to invest years of research and millions of dollars to bring a single-disease gene therapy to market, said Joni L. Rutter, Ph.D., acting director of NIHs National Center for Advancing Translational Sciences.

BGTC hopes to change that paradigm. The consortium wants to start with a common gene delivery vector known as the adeno-associated virus (AAV). Its considered one of the most effective gene delivery platforms for many human diseases.

The partnership said it will support a series of research projects and clinical trials to create new tools for AAV clinical development and regulatory evaluation. The BGTC aims to make it easier, faster and less expensive to pursue bespoke therapies in order to incentivize more companies to invest in this space and bring treatments to patients, Rutter pointed out.

The goal, over time, is to find ways to cut up-front gene therapy development costs, standardize the technology, and make it available for a broader range of diseases. The BCTC program will create a universal set of analytical tests to speed up gene and vector manufacturing. And the consortium will look for ways to streamline the regulatory framework.

By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them the most, said Peter Marks, M.D., PhD., director of the FDAs Center for Biologics Evaluation and Research.

The consortium members will contribute about $76 million over the next five years to back its projects. BGTC said it will fund research for between four and six clinical trials each focused on a different rare, single-gene disease for which no gene therapies or commercial programs currently exist. Three disease areas are targeted: Lupus, Alzheimers and Type 2 diabetes.

People with terrible genetic disorders are in dire need of solutions, Collins said. The BGTC promises to transform the field of gene therapy so we can treat, or even cure rare diseases for which no current therapy exists.

North Carolina, with its substantial biotechnology and academic resources, has become a prominent incubator for leading-edge biotechnology startups. Gene therapy is a fast-developing area within our states biotech infrastructure, said Sara Imhof, Ph.D., senior director of precision health for the North Carolina Biotechnology Center. Our related ecosystem is well positioned to contribute to and benefit from the Bespoke Gene Therapy Consortium. Its an exciting time, both for the patients who desperately need the benefits gene therapy can provide and for our innovators and industry leaders who are dedicated to this important area of science.

Original post:
Rare disease consortium is good news for NC gene therapy companies - WRAL TechWire

Wilmot Cancer Institute researchers are a step closer to understanding the complex gene interactions that cause a cell to become malignant. In a new Cell Reports study published today, the group used network modeling to hone in on a set of such interactions that are critical to malignancy, and likely to be fertile ground for broad cancer therapies.

Discrete genetic mutations that can be targeted by drugs have only been identified for a small fraction of cancer types. But those mutations rely on a downstream network of non-mutated genes in order to cause cancer. Those downstream genes and their intricate interactions may be common across many cancers and could offer a giant leap forward in cancer therapy.

One of the lead authors of the study, Hartmut Hucky Land, Ph.D., who is the deputy director of the Wilmot Cancer Institute and the Robert and Dorothy Markin Professor of Biomedical Genetics at the University of Rochester Medical Center and has worked to identify common core features of cancers for over 10 years. His goal is to find cancers shared vulnerabilities and exploit them.

Targeting non-mutated proteins that are essential to making cells cancerous is a broader approach that could be used in multiple cancers, said Land, but its hard to find these non-mutated, essential genes.

That is why Land turned to Matthew McCall, Ph.D., MHS, a Wilmot Cancer Institute investigator who is an associate professor of Biostatistics and Computational Biology at URMC, for collaboration. McCall, who is the other lead author of the study, developed a new network modeling method, called TopNet, that the group paired with genetic experiments in cells and mice to pinpoint functionally relevant gene networks.

Lands group previously identified a very diverse set of non-mutated genes that are crucial to cancer. In this study, the group wanted to see how those genes interact starting with a subset of 20 genes. Increasing or decreasing the expression of one gene in cultured cells would have numerous effects on the expression levels of the other genes in the set.

There were so many interactions, you could waste a lot of time, energy and money testing interactions that might not be useful, McCall said. To hone in on the interactions that are more likely to be useful, we used network modeling, and compared our model networks back to the lab findings, McCall said.For context, the number of possible gene network models considered by TopNet was many times greater than the estimated number of atoms in the universe. After weeding out models that didnt closely fit the observed data and further focusing in on gene interactions that appeared in at least 80 percent of the models, the team was left with a manageable set of 24 high-confidence gene interactions. Subsequent experiments demonstrated that these interactions often play an important role in malignancy.

Dr. McCalls elegant and mind-boggling methodology is essentially helping us disentangle a hairball of genetic networks, said Land. These networks are usually very messy and its nearly impossible to extract useful information from them. But Dr. McCall has found a way to cut through this Gordian knot.

The group has already tested a sampling of the genetic interactions revealed by TopNet, and confirmed via experiments in cells and mice that the interactions are functionally linked. Next, the group intends to test the limits of TopNet, with the intent to use this method to find potential cancer therapies that are broadly effective.

This work was completed as part of a $6.3M National Cancer Institute Outstanding Investigator Award granted to Land in 2015 and a K99/R00 grant from the National Human Genome Research Institute to McCall. Helene McMurray, Ph.D., assistant professor of Biomedical Genetics and Pathology and Laboratory Medicine at URMC was the first author of the study.

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Honing in on Shared Network of Cancer Genes - URMC

DUBLIN, Dec. 21, 2021 /PRNewswire/ -- The "Global Advanced Therapy Medicinal Products CDMO Market Size, Share & Trends Analysis Report by Product (Gene Therapy, Cell Therapy, Tissue Engineered), Phase, Indication, Region, and Segment Forecasts, 2021-2028" report has been added to ResearchAndMarkets.com's offering.

The global advanced therapy medicinal products CDMO market size is expected to reach USD 12.9 billion by 2028, according to the report. It is expected to expand at a CAGR of 12.0% from 2021 to 2028.

The advanced therapy medicinal products are a group of biological products for human use that involve gene therapy products, cell therapy products, and tissue-engineered products. The growth of the market is credited to the increasing clinical trials of ATMP and the rising awareness and belief among researchers regarding the benefits of advanced therapy. The COVID-19 pandemic has significantly disrupted the cell and gene therapy industry due to the complexity in the manufacturing process.

The COVID-19 pandemic has adversely affected the overall medical industry, but the pandemic boosted the operations and development of advanced therapy due to the high requirement of the products such as mesenchymal stromal cells (MSCs) for the treatment of the virus. The regulations put forward by the FDA and government authorities have created a safe environment for the healthcare workers and allowed emergency approval for the supply of essential raw materials and faster development of the vaccines and other therapy products.

Technological advancement has been a major part of tissue engineering in the last few years. This method helps to replace or restore the injured tissues and organ function. Similarly, gene and cell therapy is attracting many patients for the treatment of rare diseases, the cases of which are augmenting globally.

Advanced Therapy Medicinal Product CDMO Market Report Highlights

Key Topics Covered:

Chapter 1 Methodology and Scope

Chapter 2 Executive Summary

Chapter 3 Advanced Therapy Medicinal Products CDMO Market: Variables, Trends, & Scope3.1 Market Segmentation and Scope3.2 Market Dynamics3.2.1 Market Driver Analysis3.2.1.1 Rising number of clinical trials for ATMP3.2.1.2 Increasing outsourcing activities3.2.1.3 Growing awareness of the treatment3.2.2 Market Restraint Analysis3.2.2.1 Stringent regulatory approvals3.2.2.2 High cost of outsourcing3.3 Penetration & Growth Prospect Mapping3.4 Advanced Therapy Medicinal Products CDMO: Market Analysis Tools3.4.1 Industry Analysis - Porter's3.4.1.1 Porter's Five Forces Analysis3.4.2 PESTEL Analysis

Chapter 4 Advanced Therapy Medicinal Products CDMO Market: Product Estimates4.1 Market Share Analysis, 2020 & 20284.2 Gene Therapy4.2.1 Gene therapy market, 2016 - 2028 (USD Billion)4.3 Cell Therapy4.3.1 Cell therapy market, 2016 - 2028 (USD Billion)4.4 Tissue Engineered4.4.1 Tissue engineered market, 2016 - 2028 (USD Billion)4.5 Others4.5.1 Market, 2016 - 2028 (USD Billion)

Chapter 5 Advanced Therapy Medicinal Products CDMO Market: Phase Estimates5.1 Market Share Analysis, 2020 & 20285.2 Phase I5.2.1 Phase I market, 2016 - 2028 (USD Billion)5.3 Phase II5.3.1 Phase II market, 2016 - 2028 (USD Billion)5.4 Phase III5.4.1 Phase III market, 2016 - 2028 (USD Billion)5.5 Phase IV5.5.1 Phase IV market, 2016 - 2028 (USD Billion)

Chapter 6 Advanced Therapy Medicinal Products CDMO Market: Indication Estimates6.1 Market Share Analysis, 2020 & 20286.2 Oncology6.2.1 Oncology market, 2016 - 2028 (USD Billion)6.3 Cardiology6.3.1 Cardiology market, 2016 - 2028 (USD Billion)6.4 Central Nervous System6.4.1 Central nervous system market, 2016 - 2028 (USD Billion)6.5 Musculoskeletal6.5.1 Musculoskeletal market, 2016 - 2028 (USD Billion)6.6 Infectious Disease6.6.1 Infectious disease market, 2016 - 2028 (USD Billion)6.7 Dermatology6.7.1 Dermatology market, 2016 - 2028 (USD Billion)6.8 Endocrine, Metabolic, Genetic6.8.1 Endocrine, metabolic, genetic market, 2016 - 2028 (USD Billion)6.9 Immunology & inflammation6.9.1 Immunology & inflammation market, 2016 - 2028 (USD Billion)6.10 Ophthalmology6.10.1 Ophthalmology market, 2016 - 2028 (USD Billion)6.11 Haematology6.11.1 Haematology market, 2016 - 2028 (USD Billion)6.12 Gastroenterology6.12.1 Gastroenterology market, 2016 - 2028 (USD Billion)6.13 Others6.13.1 Others market, 2016 - 2028 (USD Billion)

Chapter 7 Advanced Therapy Medicinal Products CDMO Market: Regional Analysis

Chapter 8 Company Profiles8.1 Strategic Framework8.2 Company Profiles8.2.1 Celonic8.2.1.1 Company Overview8.2.1.2 Financial performance8.2.1.3 Product Benchmarking8.2.1.5 Strategic Initiatives8.2.2 Bio Elpida8.2.2.1 Company Overview8.2.2.2 Financial performance8.2.2.3 Product Benchmarking8.2.2.6 Strategic Initiatives8.2.3 CGT Catapult8.2.3.1 Company Overview8.2.3.2 Financial performance8.2.3.3 Product Benchmarking8.2.3.6 Strategic Initiatives8.2.4 Rentschler Biopharma SE8.2.4.1 Company Overview8.2.4.2 Financial performance8.2.4.3 Product Benchmarking8.2.4.6 Strategic Initiatives8.2.5 AGC Biologics8.2.5.1 Company Overview8.2.5.2 Financial performance8.2.5.3 Product Benchmarking8.2.5.6 Strategic Initiatives8.2.6 Catalent8.2.6.1 Company Overview8.2.6.2 Financial performance8.2.6.3 Product Benchmarking8.2.6.6 Strategic Initiatives8.2.7 Lonza8.2.7.1 Company Overview8.2.7.2 Financial Performance8.2.7.3 Product Benchmarking8.2.7.5 Strategic Initiatives8.2.8 WuXi Advanced Therapies8.2.8.1 Company Overview8.2.8.2 Financial performance8.2.8.3 Product Benchmarking8.2.8.5 Strategic Initiatives8.2.9 BlueReg8.2.9.1 Company Overview8.2.9.2 Financial performance8.2.9.3 Product Benchmarking8.2.9.6 Strategic Initiatives8.2.10 Minaris Regenerative Medicine8.2.10.1 Company Overview8.2.10.2 Financial performance8.2.10.3 Product Benchmarking8.2.10.5 Strategic Initiatives8.2.11 Patheon8.2.11.1 Company Overview8.2.11.2 Financial performance8.2.11.3 Product Benchmarking8.2.11.5 Strategic Initiatives

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Outlook on the Advanced Therapy Medicinal Products CDMO Global Market to 2028 - Rising Number of Clinical Trials for ATMP is Driving Growth -...

SAN RAFAEL, Calif. and SHANGHAI, Dec. 16, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, today announced a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases.

The partnership will leverage Skyline Therapeutics' integrated AAV gene therapy platform based on its proprietary vector engineering and design technology and manufacturing capability to develop innovative gene therapies with a focus on genetic dilated cardiomyopathies (DCM), a group of progressively advancing, devastating diseases with no targeted treatment options.

Under the agreement, BioMarin and Skyline Therapeutics will collaborate on discovery and research through to an Investigational New Drug Application (IND). BioMarin brings experience in gene therapy development, cardiovascular biology and insights into genetic basis of diseases, and Skyline contributes its expertise in developing gene therapy products including vector engineering and design technology and manufacturing capabilities to this collaboration. Each company will advance the programs through clinical development in their pre-defined territories.

In support of its R&D efforts for the collaborative projects, Skyline Therapeutics will receive an undisclosed payment associated with signing, comprising an upfront payment and an equity investment from BioMarin, and is eligible to receive pre-specified payments for R&D, regulatory and commercial milestones.

BioMarin will have the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America, and Skyline Therapeutics will be responsible for commercialization in the Asia-Pacific region. In addition, Skyline Therapeutics will be eligible to receive royalty payments on future sales from BioMarin in its territories.

"We are thrilled to announce what we anticipate will be a fruitful collaboration at the interface between Skyline's innovative approach to AAV vector engineering and design and our team's proven expertise in creating and developing gene therapies," said Kevin Eggan, Group Vice President, Head of Research and Early Development, from BioMarin.

"We are excited to partner with Skyline Therapeutics to tackle these genetic forms of dilated cardiomyopathy. This collaboration strengthens our leadership in cardiac gene therapy and extends our R&D collaboration to Asia, where a large number of patients suffer from these devastating diseases," said Brinda Balakrishnan, Group Vice President, Corporate and Business Development at BioMarin. "We look forward to fostering this collaboration and bringing transformative medicines to patients worldwide."

"Dilated cardiomyopathy is a serious cardiac disorder in which structural or functional abnormalities of the heart muscle can lead to complications such as arrhythmia and heart failure, resulting in substantial morbidity and mortality. Mutations in many genes are associated with the development of DCM, among other etiologies for the disease," said Jay Hou, Chief Scientific Officer at Skyline Therapeutics. "Together with BioMarin's team we have identified a number of critical genes associated with DCM. We are delighted to work closely with BioMarin and apply our AAV vector technology to interrogate these new targets and develop novel treatments for DCM patients."

"The collaboration with BioMarin leverages both companies' capabilities in the development of gene therapies. With the BioMarin team, we share the goal of working in concert to develop therapies for genetic cardiovascular disease that address high unmet medical needs," said Amber Cai, CEO of Skyline Therapeutics. "Together, we will utilize gene therapy to tackle cardiovascular diseases with a disease modifying trailblazing approach that could change the treatment paradigm in these conditions."

About Dilated Cardiomyopathy (DCM)

DCM is a common cause of heart failure and end-stage DCM, which often leads to heart transplantation. Despite improvements in pharmacotherapy and care, the five-year survival rate of DCM is only about 50%. Hundreds of thousands of patients suffer from the genetic forms of DCM in U.S., EU, China, and Japan. More than 50 genes associated with DCM have been identified, accounting for 40-50% of familial DCM cases. Many of these genes encode proteins with important known functions in cardiomyocytes related to cytoskeletal, sarcomere and nuclear envelope biology. Our aim is to correct the pathways altered by these genetic contributors to DCM through AAV based gene therapy, in each case addressing the root cause of the disease.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit http://www.biomarin.com.Information on such website is not incorporated by reference into this press release.

About Skyline Therapeutics

Skyline Therapeutics is a fully integrated gene and cell therapy company dedicated to the discovery, development and delivery of innovative therapies. Established in 2019, Skyline Therapeutics has built a proprietary AAV-based gene therapy platform that integrates novel capsid engineering and vector design, analytical and process development, and state-of-the-art GMP manufacturing capabilities that support large scale clinical-grade vector production. The Skyline team of world-class experts and leaders in science, technology and business brings industry-leading know-how and is advancing a pipeline of diversified programs that address multiple diseases including ocular, neurological, metabolic and blood disorders. Skyline Therapeutics is also broadening its therapeutic expertise to cover more disease areas with high unmet need such as cardiovascular disorders through strategic partnerships. Headquartered in China, Skyline Therapeutics currently has research, development and manufacturing capabilities in Shanghai and Hangzhou. http://www.skytx.com

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: expectations related to themulti-year global strategic collaboration with Skyline for the discovery, development and commercialization of AAV gene therapies for dilated cardiomyopathy and pre-specified payments to Skyline for R&D, regulatory and commercial milestones, and the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. Additional important factors to be considered in connection with forward-looking statements are detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

References

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

Felisa Feng

Skyline Therapeutics

[emailprotected]

SOURCE BioMarin Pharmaceutical Inc.

http://www.biomarin.com

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BioMarin and Skyline Therapeutics Announce Strategic Collaboration to Develop Novel Gene Therapies for Cardiovascular Diseases - PRNewswire

Paris (AFP) Camille Abbey was two months away from giving birth to twins when she sensed something wasn't right.

"I felt strange all week. I had swelled enormously. I had a lot of trouble moving," said the 33-year-old French journalist.

Abbey's midwife found her blood pressure spiking and sent her to hospital where doctors confirmed she had preeclampsia, a potentially life-threatening complication.

The risks of preeclampsia have been known about for centuries, but there is still no cure or prevention and a lack of awareness remains a problem.

"Although one in every 12 pregnancies are affected by preeclampsia, many pregnant women have never heard of the disease," Patricia Maguire, director of the Institute for Discovery at University College Dublin, told AFP.

Doctors made a breakthrough 10 years ago that allowed them to develop the first diagnostic test, and Maguire is working on a second one, with trials under way in Ireland.

Early diagnosis is lifesaving because the condition sets in without symptoms.

Once they appear, the only way to stop complications like liver and kidney failure, or death, is to deliver the baby -- even if it is premature.

A 2021 study showed that hypertensive disorders of pregnancy have increased in the last nearly 30 years, though better screening and population growth may account for the rise.

The study in the BMC Pregnancy and Childbirth journal also said that most deaths occur in low-income settings where doctors say the toll is likely higher than reported.

An estimated 76,000 women and 500,000 babies die every year due to hypertensive pregnancy conditions like preeclampsia and eclampsia, a rare complication, according to a 2015 study in the Journal of Family Medicine and Primary Care.

Sarah Kilpatrick, an obstetrician-gynecologist specialising in preeclampsia at Cedars-Sinai hospital in Los Angeles, says educating women is crucial.

"The hard part for women is many times they feel fine," Kilpatrick told AFP, "so you can't even believe you have something like preeclampsia."

- Like 'lightning' -

Emmanuelle Honore survived eclampsia -- named after the Greek word for lightning -- which can develop if preeclampsia goes undetected and lead to potentially fatal seizures.

Her decision to walk herself to hospital after monitoring her own blood pressure saved her life.

"They didn't wait for the blood test results to come back to decide to do an emergency cesarean," said the Paris-based archaeologist, 37.

Immediately after giving birth, she began to convulse.

"It was a question of minutes. The baby and I were incredibly lucky," she told AFP. "During the seizure I felt myself leaving my own body."

Today, her son is healthy and turns three in February.

But the eclampsia has left Honore psychologically scarred and unable to give birth again.

"For me, there is a before and an after -- it's two different lives."

The world's first diagnostic test, developed by kidney specialists Ananth Karumanchi and Ravi Thadhani by linking the levels of two proteins to the disorder's onset, has been available in Europe for a decade.

It is in clinical trials in the United States, where preeclampsia disproportionately affects Black women due to a higher prevalence of risk factors like diabetes, obesity and stress, the American Heart Association says.

"(The trials) include the diversity of women in the United States and by the end of this year we will be able to determine whether the diagnostics have just as much benefit and specificity as in Europe," Thadhani told AFP.

The test developed by Maguire not only detects preeclampsia with a blood sample, it uses artificial intelligence to provide a timeframe for safe delivery, allowing as much time as possible for the foetus to develop.

An article published in May said the test had the potential to be streamlined so that kits could be sent to hospitals with easy-to-collect samples analysed using online information.

"The big dream is to reach every person who needs this test across the world," Maguire said.

Even after labour, preeclampsia sufferers are not necessarily out of danger.

Kilpatrick said about one in 10 women whose preeclampsia was their first time with high blood pressure need to stay on anti-hypertensive medication after giving birth.

When her twins were born in October 2020, Abbey thought her ordeal was over but her blood pressure failed to go back to normal.

She was later diagnosed with the dangerous preeclampsia-related condition HELLP (Hemolysis, Elevated Liver enzymes and Low Platelets) syndrome, which affects some 15 percent of women with severe preeclampsia.

While her premature babies received state-of-the-art care, Abbey said she stayed in a post-op recovery room for days where doctors could do little more than monitor her vital signs and try to control her blood pressure.

Vassilis Tsatsaris, an obstetrician-gynecologist at Paris' Port Royal hospital, said research on finding treatments for preeclampsia and other complications has been impeded by the risks for the unborn baby.

"We live in an era where therapeutic innovations for cancer, vaccines or gene therapy are advancing very, very quickly," he told AFP.

"Unfortunately when it comes to pregnancy, things move much more slowly."

2021 AFP

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Preeclampsia: an ongoing battle to save lives - FRANCE 24

BEIJING & CAMBRIDGE, Mass., November 01, 2021--(BUSINESS WIRE)--CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that it has entered into a two-year sponsored research agreement with the University of Washington School of Medicine, in Seattle, Washington, for gene therapy research in Duchenne muscular dystrophy (DMD), a rare neuromuscular disease. The program will be under the direction of Jeffrey Chamberlain, Ph.D., professor in the Departments of Neurology, Medicine and Biochemistry, the McCaw Endowed Chair in Muscular Dystrophy at the University of Washington School of Medicine, and Director of the Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center of Seattle. Guy Odom, Ph.D., Research Assistant Professor in the Department of Neurology at the University of Washington, will serve as the co-principal investigator.

Dr. Chamberlain is internationally renowned as a pioneer and one of the top researchers in the field of gene therapies for muscle diseases. His lab has been studying muscular dystrophy mechanisms, particularly dystrophin structure, and gene therapy approaches. They were the first to show that adeno-associated virus (AAV) vectors could be used for systemic gene delivery to muscle.

"We are thrilled to enter into this research agreement with Dr. Chamberlain, who has been leading the world in DMD research for decades, as we advance our gene therapy research program in neuromuscular disorders," said James Xue, Ph.D., Founder, Chairman and CEO, CANbridge Pharmaceuticals Inc. "Duchenne muscular dystrophy is the most common of the hereditary neuromuscular diseases and, despite recent approvals for exon-skipping therapies, remains severely underserved medically. We believe that the best gene therapy for this devastating disease has not yet been discovered, and we look forward to working with Dr. Chamberlain and his team on their innovative research, as well as the new treatments that may arise from it."

Story continues

About Dystrophinopathies

Duchenne muscular dystrophy (DMD) is a rare muscle disorder, but it is one of the most frequent genetic conditions that primarily affects males. DMD usually presents in early childhood and is characterized by rapidly progressive muscle degeneration and weakness, leading to loss of ambulation by about 12 years of age. Cardiomyopathy is a common cause of morbidity and death in DMD patients. The incidence of DMD is estimated to be 1/3,500 1/5,000 male births worldwide and 1/4,560 in China, according to the National Organization for Rare Disease and published peer review.

About the Chamberlain Laboratory, University of Washington Department of Neurology

The lab is focused on muscular dystrophy research with two major goals: to develop a better understanding of the molecular basis of the pathophysiology of the diseases, and to develop gene and cell therapies that will correct and treat the muscular dystrophies. Major targets for therapy include Duchenne muscular dystrophy and LGMD2I.

About the Odom Laboratory, University of Washington Department of Neurology

The lab is focused on developing a more thorough understanding of the inherent muscle biology occurring during muscular dystrophy disease progression. As such, the labs research generally involves developing or improving genetic-based therapies.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies.

CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology.

These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme (GBM).

CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Medical School (UMass) and LogicBio.

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

View source version on businesswire.com: https://www.businesswire.com/news/home/20211101005332/en/

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CANbridge Pharmaceuticals Inc. ir@canbridgepharma.com

Media: Deanne Eagle Planet Communications deanne@planetcommunications.nyc 917.837.5866

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CANbridge Pharmaceuticals Enters into Duchenne Muscular Dystrophy Gene Therapy Research Agreement with University of Washington School of Medicine -...

Levodopa, the commonly prescribed dopamine-restoring drug for Parkinson's disease, loses its effectiveness over time. Researchers at Northwestern University say they've found a potential method forreviving the drug's benefits: gene therapy.

The researchers restored the ability of neurons to convert levodopa into dopamine in mice with a gene therapy that targets the substantia nigra region of the brain. By effectively recreating a healthy environment in the brain, the therapy eliminated abnormal brain activity that causes movement difficulties in Parkinson's patients, the teamreported in Nature.

The new findings also provided insights into why dopamine-releasing neurons wither away in Parkinson's. By studying the genetic features of theneurons in Parkinson's models, the Northwestern researchers showed that damage to the mitochondria, the power suppliers inside of dopamine-producing neurons, triggers events that lead to Parkinson's.

"Whether mitochondrial damage was a cause or consequence of the disease has long been debated. Now that this issue is resolved, we can focus our attention on developing therapies to preserve their function and slow the loss of these neurons," said James Surmeier, Ph.D., chair of neuroscience at Northwestern's Feinberg School of Medicine, in a statement.

Theinsights could be usedto develop tests that identify Parkinson'sin people five to 10 years before it manifests, Surmeier suggested.

RELATED:Neurocrine exits $165M Parkinson's pact with Voyager after FDA hold

Efforts to develop gene and cell therapies for Parkinson's are underway, with mixed results so far.Bayer has started two early-stage trials: a gene therapy being developed byits subsidiary AskBio and a stem cell treatment from its unit BlueRock Therapeutics.

Voyager Therapeutics has suffered several setbacks in its efforts to develop a gene therapy for Parkinson's.Sanofi ended its deal with Voyager in October 2017, AbbVie nixed its pact in August 2020 and Neurocrine Biosciences axed its tie-up in February of this year after a clinical hold was placed on a phase 2 trial last December. Pfizer inkeda $630 million pact with Voyager last month to use its capsids in neurologic and cardiovascular gene therapies, though the specific disease targets were not disclosed.

Other researchers are also looking for innovative ways to spruce up dopamine-producing neurons. A team at the University of San Diego, California developeda gene therapy technique that turned astrocyte cells into dopamine-producing neurons, for example.

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Parkinson's gene therapy restores responses to dopamine-boosting drug in mouse models - FierceBiotech

image:Alliance for Cancer Gene Therapy (ACGT) Scientific Advisory Council Chair Michael T. Lotze, MD, of the University of Pittsburgh, will be honored with the 2021 Society for Immunotherapy of Cancer (SITC) Lifetime Achievement Award at the SITC 36th Annual Meeting, Nov. 10-14, in Washington D.C. view more

Credit: University of Pittsburgh

Alliance for Cancer Gene Therapy (ACGT) Scientific Advisory Council Chair Michael T. Lotze, MD, of the University of Pittsburgh, will be honored with the 2021 Society for Immunotherapy of Cancer (SITC) Lifetime Achievement Award at the SITC 36th Annual Meeting, Nov. 10-14, in Washington D.C.

Dr. Lotze is currently Chief Cellular Therapy Officer of Nurix Therapeuticsand professor of surgery, immunology and bioengineering at the University of Pittsburgh School of Medicine. He is widely regarded as the leader in exploring cancer as a disorder of cell death and is devising novel strategies to approach the disease in this context. He initiated the first approved gene therapy protocols at the National Institutes of Health and has treated more than 100 patients on gene therapy protocols at the University of Pittsburgh. He is the co-inventor of 10 patents in dendritic cell vaccines and antigen discovery, and author of more than 500 scientific papers and chapters in basic and applied tumor immunology and cytokine biology.

Dr. Lotze leads the 14 members of the ACGT Scientific Advisory Council in rigorously reviewing and monitoring the research selected for funding by ACGT. The importance and value that the Council contributes to the ACGT funding process distinguishes ACGT from many other funding agencies. Council members are among the most accomplished thought-leaders in the field of cancer cell and gene therapy. They are experienced scientists whose decades of research and patient care have elevated them into important leadership positions at top institutions across the U.S. and in Canada.

Society for Immunotherapy of Cancer is a member-driven organization dedicated to improving cancer patient outcomes by advancing the science and application of cancer immunotherapy through educational programs that foster scientific exchange and collaboration. Learn more about SITC at http://www.sitcancer.org.

Alliance for Cancer Gene Therapy

For more than 20 years, Alliance for Cancer Gene Therapy (ACGT) has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. The organization funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. 100% of all public funds raised directly support research and programs. For more information, visitacgtfoundation.org, call (203) 358-5055,or join the Alliance for Cancer Gene Therapy community onFacebook,Twitter,LinkedIn, Instagram andYouTube @acgtfoundation.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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ACGT Scientific Advisory Council Chair Michael T. Lotze, MD, honored with Lifetime Achievement Award from Society for Immunotherapy of Cancer -...

PALO ALTO, Calif., Nov. 3, 2021 /PRNewswire/ --BridgeBioPharma, Inc. (Nasdaq: BBIO),a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational adeno-associated virus (AAV) 9 gene therapy for the treatment of Canavan disease. Canavan disease is an ultra-rare and fatal disease that affects approximately 1,000 children in the United States and European Union. There are currently no approved therapies for the condition.

"Dosing the first patient in our Canavan disease trial is a significant achievement for our gene therapy team and we would not be here without the support of our collaborative partners in the patient, medical and scientific communities," said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. "We are committed to the Canavan community and will work to advance this trial and the other programs in our gene therapy portfolio because we believe deeply in the potential of gene therapy to improve and save lives."

"Based on the efficacy and safety data we've observed in our preclinical studies, we are hopeful that our investigational gene therapy can become a meaningful treatment option for children living with Canavan. Right now, these children only have access to supportive care because there are currently no approved therapies to treat this devastating disease," added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy.

The Phase 1/2 open-label study is designed to evaluate the safety, tolerability, and pharmacodynamic activity of the company's AAV9 gene therapy, BBP-812, in pediatric patients with Canavan disease. In the initial dose-finding phase of the study, each patient will receive a single intravenous (IV) infusion of BBP-812. The primary outcomes of the study are safety, as well as change from baseline of urine and central nervous system N-acetylaspartate (NAA) levels. Motor function and development will also be assessed. Preclinical proof-of-concept data have shown the approach restores survival and normal motor function.

"Gene therapy is designed to treat diseases at their source, which for Canavan disease would be an extremely beneficial treatment option. Through this trial, we hope to provide evidence that this investigational therapy could represent a promising treatment option for Canavan patients and their families. We are grateful to the first family for participating," said Florian Eichler, M.D., director of the Leukodystrophy Service and principal investigator at Massachusetts General Hospital, the first clinical site open in the CANaspire trial.

BridgeBio's gene therapy was originally developed by Guangping Gao, Ph.D., and Dominic J. Gessler, M.D., Ph.D., at the University of Massachusetts Medical School. Dr. Gao, a pioneer in AAV gene therapy, was also the first person to clone the ASPA gene, which in its mutated form causes Canavan disease. Dr. Gao has been working on developing a cure for Canavan disease for more than 25 years.

"Throughout my career, I've been passionate about connecting with families in the Canavan community to understand how to help them through their journey. After decades of research and work, I'm thrilled that we have dosed the first patient in this trial. I'm hopeful that this represents a meaningful turning point for the Canavan community," said Dr. Gao, co-director of the Li Weibo Institute for Rare Diseases Research, director of the Horae Gene Therapy Center and Viral Vector Core, and professor at University of Massachusetts Medical School.

BridgeBio's investigational AAV9 gene therapy for Canavan disease is one of the Company's 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and genetically-driven cancers. An initial Phase 1/2 data readout for Canavan disease is expected in 2022. For more information about the CANaspire trial, visit TreatCanavan.com or ClinicalTrials.gov (NCT04998396).

About BBP-812BBP-812 is an investigational AAV9 gene therapy for Canavan disease. Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, potentially correcting the disease at its source. Preclinical proof-of-concept results in Canavan disease models have shown the approach restores survival and normal motor function. BBP-812 was granted Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation by the U.S. Food and Drug Administration. BBP-812 was also granted Orphan Drug Designation by the European Medicines Agency.

About Canavan DiseaseAffecting approximately 1,000 children in the United States and European Union, Canavan disease is an ultra-rare, disabling and fatal disease with no approved therapy. Most children are not able to meet developmental milestones, are unable to crawl, walk, sit or talk, and pass away at a young age. The disease is caused by an inherited mutation of the ASPA gene, which codes for aspartoacylase, a protein that breaks down a compound called N-acetyl-L-aspartic acid (NAA). Deficiency of aspartoacylase activity results in accumulation of NAA, and ultimately results in toxicity to myelin in ways that are not well understood. Myelin insulates the nerves, and without it, neurons are unable to send and receive messages as they should. The current standard of care is limited to supportive therapy.

About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio's pipeline of over 30 development programs ranges from early science to advanced clinical trials, and its commercial organization is focused on delivering the company's first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.comand follow us onLinkedInandTwitter.

BridgeBio Pharma, Inc. Forward-Looking StatementsThis press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the "Securities Act"), and Section 21E of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to the timing and success of BridgeBio's Phase 1/2 clinical trial of BBP-812 for the treatment of Canavan disease, expectations, plans and prospects regarding BridgeBio's regulatory approval process for BBP-812, the ability of BBP-812 to treat Canavan disease in humans, and the timing and success of initial top-line Phase 1/2 date of BBP-812, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, BridgeBio's ability to continue and complete its Phase 1/2 clinical trial of BBP-812 for the treatment of Canavan disease, past data from preclinical studies not being indicative of future data from clinical trials, BridgeBio's ability to advance BBP-812 in clinical development according to its plans, the ability of BBP-812 to treat Canavan disease, the ability of BBP-812 to retain Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency, and potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; as well as those set forth in the Risk Factors section of BridgeBio's most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent SEC filings, which are available on the SEC's website atwww.sec.gov.Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact: Grace Rauh[emailprotected](917) 232-5478

BridgeBio Investor Contact: Katherine Yau[emailprotected](516) 554-5989

SOURCE BridgeBio

https://bridgebio.com/

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BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Canavan Disease - PRNewswire

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) --Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform.We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200

Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent Business Updates - BioSpace

NEW YORK and BASEL, Switzerland, Nov. 4, 2021 /PRNewswire/ --Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, announced that an abstract demonstrating the clinical benefit of the company's lead product candidate ARU-1801 has been published online and will be the subject of a poster presentation at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place in Atlanta, Georgia fromDecember 11 to 14, 2021.Punam Malik, M.D., Director of the Cincinnati Comprehensive Sickle Cell Center and Program Leader of the Hematology and Gene Therapy Program at the Cincinnati Children's Hospital Medical Center, will present the data at6:00 to 8:00 PM ESTonDecember 13, 2021.

"Clinical data from our ongoing MOMENTUM study has shown 100 percent resolution of vaso-oclusive events (VOE) in our recently treated SCD patients at 18 and 12 months of follow up," said Will Chou, MD, Aruvant chief executive officer. "In addition, we are excited to be sharing additional clinical data at ASH that demonstrates how the unique anti-sickling potency of ARU-1801 translates to these robust clinical outcomes."

Dr. Malik will present data from theongoing MOMENTUM study, an open label Phase 1/2 clinical trial examining ARU-1801 as a one-time potentially curative gene therapy for individuals with sickle cell disease (SCD). The MOMENTUM study examines ARU-1801, an autologous lentiviral cell therapy with a modified, highly potent gamma globin payload, in individuals with severe SCD. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy. ARU-1801 is designed to address the limitations of current curative allogeneic transplant options, such as low donor availability, the risk of graft-versus-host disease and toxicity from myeloablative chemotherapy. The data to be presented at ASH highlights clinically meaningfulreduction in participants' VOEs and the unique attributes that contribute to the potency of ARU-1801.

"The emerging clinical data shows that ARU-1801 holds promise for achieving durable responses in patients with severe SCD using only reduced intensity conditioninga key differentiator from other investigational gene therapy regimens," said Dr. Malik. "Given the advantage for patients, providers and payers a reduced conditioning regimen offers, ARU-1801 has the potential to be an important option for SCD patients seeking gene therapy, including those in low resource settings."

Abstract and Poster Presentation Information

Title: Safety and Efficacy of ARU-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 MOMENTUM Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning

Publication Number: 3970Session Name: 801. Gene Therapies: Poster IIIDate:Monday, December 13, 2021Presentation Time:6:00 to 8:00 PM ESTLocation: Georgia World Congress Center, Hall B5 or online through the ASHwebsite.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromSCD. ARU-1801, an investigational lentiviral gene therapy, is being studied in aPhase 1/2 clinical trial,the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. The company's second product candidate, ARU-2801, is in development to cure hypophosphatasia, a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high morbidity and mortality when not treated. Data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival. For more information on the ongoing ARU-1801 clinical study, please visit http://www.momentumtrials.com,and for more on the company, pleasevisitwww.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

SOURCE Aruvant Sciences

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Aruvant Announces ARU-1801 Data to be Presented at the 63rd American Society of Hematology (ASH) Annual Meeting - PRNewswire

BRISBANE, Calif.--(BUSINESS WIRE)-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today reported third quarter financial results and provided business and clinical highlights.

We are delighted to share clinical data and business updates across several programs demonstrating that Sangamo has three important assets progressing toward late-stage development. Our gene therapy portfolio is advancing with accumulating safety and efficacy data in our Fabry and hemophilia A programs, and preliminary proof-of-concept data demonstrate the clinical potential of our zinc finger genome engineering technology in sickle cell disease. These data readouts show the progression of our first-generation genomic medicine pipeline and potentially pave the way for new treatments. Our next generation programs focus on genome regulation and allogeneic CAR-Treg cell therapy, where we have a robust preclinical pipeline in neurological and autoimmune diseases. We are energized by this momentum and look forward to continued execution of our corporate strategy, said Sandy Macrae, Chief Executive Officer of Sangamo.

Recent Clinical and Business Highlights

Fabry Disease First four patients dosed exhibited above normal -Gal A activity; Phase 3 planning initiated

Sickle Cell Disease Preliminary-proof-of-concept data will be presented at ASH as clinical program advances

Hemophilia A Four patients at highest dose experienced mean FVIII activity of 30.9% at week 104

Renal Transplant First patient enrolled, expect two patients to be dosed by mid-2022

Research, Manufacturing, and Corporate Updates

Third Quarter 2021 Financial Results

Consolidated net loss attributable to Sangamo for the third quarter ended September 30, 2021 was $47.7 million, or $0.33 per share, compared to a net loss attributable to Sangamo of $1.6 million, or $0.01 per share, for the same period in 2020.

Revenues

Revenues for the third quarter ended September 30, 2021, were $28.6 million, compared to $57.8 million for the same period in 2020, a decrease of $29.2 million.

The reduction in revenue was primarily due to a $39.3 million decrease related to our giroctocogene fitelparvovec and C9ORF72 collaboration agreements with Pfizer, resulting from the completion of our activities in 2020, and a $2.3 million decrease related to our collaboration agreement with Sanofi. These decreases were partially offset by higher revenues of $11.5 million and $1.3 million related to our collaboration agreements with Novartis and Biogen, respectively.

GAAP and Non-GAAP operating expenses

2021

2020

2021

2020

$

62.5

$

45.3

$

179.0

$

128.3

14.5

16.2

47.1

50.2

77.0

61.5

226.1

178.5

(7.9

)

(6.7

)

(24.9

)

(19.1

)

$

69.1

$

54.8

$

201.2

$

159.4

Total operating expenses on a GAAP basis for the third quarter ended September 30, 2021 were $77.0 million compared to $61.5 million for the same period in 2020. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the third quarter ended September 30, 2021 were $69.1 million compared to $54.8 million for the same period in 2020.

The increase in total operating expenses on a GAAP basis was primarily driven by our higher clinical and manufacturing supply expenses along with our increased headcount to support the advancement of our clinical trials and our ongoing collaborations.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of September 30, 2021 were $519.0 million compared to $692.0 million as of December 31, 2020.

Revised Financial Guidance for 2021

We are revising our full-year operating expense guidance initially provided on February 24, 2021 and reiterated most recently on August 5, 2021 as follows:

$285 to $305

$300 to $310

$255 to $275*

$265 to $275**

Conference Call

Sangamo will host a conference call today, November 4, 2021, at 9:15 a.m. Eastern Time, which will be open to the public. The call and live Q&A will be webcast.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 5178059. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. Call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 5178059.

About Sangamo Therapeutics

Sangamo Therapeutics is a clinical-stage biopharmaceutical company with a robust genomic medicines pipeline. Using ground-breaking science, including our proprietary zinc finger genome engineering technology and manufacturing expertise, Sangamo aims to create new genomic medicines for patients suffering from diseases for which existing treatment options are inadequate or currently dont exist. For more information about Sangamo, visit http://www.sangamo.com.

Forward-Looking Statements

This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to the therapeutic and commercial potential of our product candidates, the anticipated plans and timelines of Sangamo and our collaborators for screening, enrolling and dosing patients in and conducting our ongoing and potential future clinical trials and presenting clinical data from our clinical trials, the anticipated advancement of our product candidates to late-stage development including potential future Phase 3 trials, anticipated implementation of a protocol amendment for the Phase 3 AFFINE clinical trial of giroctocogene fitelparvovec and the resumption of the dosing of additional patients in the trial; our revised 2021 financial guidance related to GAAP and non-GAAP total operating expenses and stock-based compensation; our continued execution of our corporate strategy; the anticipated completion of our in-house cell therapy manufacturing facility in Valbonne, France; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to the effects of the evolving COVID-19 pandemic and the impacts of the pandemic on the global business environment, healthcare systems and business and operations of Sangamo and our collaborators, including the initiation and operation of clinical trials; the research and development process, including the enrollment, operation and results of clinical trials and the presentation of clinical data; the uncertain timing and unpredictable nature of clinical trials and clinical trial results, including the risk that any protocol amendment for the Phase 3 AFFINE trial of giroctocogene fitelparvovec may not be accepted by the relevant review bodies in a timely manner, or at all, or that the FDA may not lift its clinical hold on the Phase 3 AFFINE trial in a timely manner, or at all, each of which could further delay or preclude further patient dosing in the trial as well as the risks that therapeutic effects observed in clinical trial results will not be durable in patients and that final clinical trial data will not validate the safety and efficacy of our product candidates; reliance on results of early clinical trials, which results are not necessarily predictive of future clinical trial results; our limited experience manufacturing biopharmaceutical products, including the risks that we may be unable to maintain compliant manufacturing facilities, build additional facilities and manufacture our product candidates as intended; and our ability to achieve expected future financial performance.

There can be no assurance that we and our collaborators will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and our collaborators. These risks and uncertainties are described more fully in our Securities and Exchange Commission filings and reports, including in our Annual Report on Form 10-K for the year ended December 31, 2020 as supplemented by our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021. Forward-looking statements contained in this announcement are made as of this date, and we undertake no duty to update such information except as required under applicable law.

Non-GAAP Financial Measure

To supplement our financial results and guidance presented in accordance with GAAP, we present non-GAAP total operating expenses, which exclude stock-based compensation expense from GAAP total operating expenses. We believe that this non-GAAP financial measure, when considered together with our financial information prepared in accordance with GAAP, can enhance investors and analysts ability to meaningfully compare our results from period to period and to our forward-looking guidance, and to identify operating trends in our business. We have excluded stock-based compensation expense because it is a non-cash expense that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. This non-GAAP financial measure is in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We encourage investors to carefully consider our results under GAAP, as well as our supplemental non-GAAP financial information, to more fully understand our business.

2021

2020

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Sangamo Therapeutics Reports Recent Business and Clinical Highlights and Third Quarter 2021 Financial Results - BioSpace