Category Archives: Gene therapy

If a new Planet of the Apes or Jurassic Park film comes out, Im going to go see it. The latest, Jurassic World: Dominion, didnt disappoint.A plague of locusts

The science is mostly accurate, the bioethics message obvious, and the plot adheres to Isaac Asimovs change one thing rule for science fiction. In the world of Jurassic Park, that lone variable is time. We just have to accept that a titanosaur like Argentinosaurus somehow grew and developed from a lab-nurtured baby to a 130-foot-tall and 110-ton adult in a few years.

The official summary from IMDb for the new film is vague and continues the impossibly-rapid-growth theme:

Four years after the destruction of Isla Nublar, dinosaurs now liveand huntalongside humans all over the world. This fragile balance will reshape the future and determine, once and for all, whether human beings are to remain the apex predators on a planet they now share with historys most fearsome creatures in a new Era.

Four years? Animals radiating around the world? Even rats or rabbits couldnt do that. And the film is actually more about insects.

Evil company Synbio has let loose hordes of genetically modified locusts, each the size of a shoebox. They eat only crops that havent been grown with the companys insecticide. (Old story, the first GMO corn was grown in 1996.) The rapidly-reproducing, gargantuan locusts are expected to decimate the nations crops by summers end.

The upper floors at Synbio appear innocent: a clearinghouse to rehab the poor reptiles roaming the planet after they burst free in the last movie. The beasts are then sent to a Wildlife Relocation Center in Pennsylvania or to a facility in the Dolomite Mountains of Italy for some R&R. Synbio bought the company behind Jurassic Park back in the 90s, and it is now focusing on saving 20 key species.

But there are other parts to the company.

The Habitat and Development Laboratory contains untouched genomes of rare species. Underground lies Synbios top secret project, hexapod. Six legs.

The company is conveniently near amber deposits that provided the initial dino DNA back in the 1990s in preserved mosquitoes, and this time around, the locust genetic material. The amber mine looks a lot like Howe Caverns, near where I live in upstate New York.

Of course other folks have dinos too. Sawridge Cattle Company in western Nevada, for example, is now an illegal breeding facility. Cute baby triceratopses are stuffed into cages like the ones at county fairs that house pigs.

The locusts and dinosaurs are the backdrop to the human drama that centers around 14-year-old Maisie Lockwood (Isabella Sermon), granddaughter of Sir Benjamin Lockwood. He was the business partner of John Hammond, both responsible for the modern-day dinos in the first place.

Maisie, it turns out, is a clone. Her mother, Charlotte (Elva Trill), wanted to have a kid, so why not just clone herself? The 23 human chromosome pairs on her computer screen announce that she is a geneticist, although Im a geneticist too and I never feel the need to remind myself of the number, which isnt useful for anything.

Young Maisie is precocious and gorgeous and for unexplained reasons, has an English accent. Owen Grady (Chris Pratt) and Claire Dearing (Bryce Dallas Howard), from the last movie, adopt her and they live in the woods to protect her from kidnappers.

A few dinosaurs live near their house, and Maisie befriends an adorable mom and her baby, Blue and Beta. They arose from dino DNA mixed with monitor lizard DNA, like the frog DNA patched into the genomes of the original Jurassic Park dinos. We need the little raptor to understand you. They are genetically identical, like you and Charlotte. Charlotte, like Blue, was able to have a child all by herself, sneers a kidnapper, for Maisie, too, arose without the contribution of a sperm.

But why would anyone want to kidnap Maisie? Why is she the most valuable IP on the planet?

When Maisie was a toddler, her mother Charlotte, much to her surprise despite the chromosomes on her screen, suddenly sickened from an unnamed, undiagnosed genetic disease, even though young-adult-onset of a genetic disease is rare. Oops! She cloned a child with a mystery mutation!

Not to worry. Charlotte whipped up some viral DNA to deliver working genes, something thats been done in gene therapy since the first experiments, in 1990, coincidentally when Michael Crichton published Jurassic Park. Back then, we could just add genes in gene therapy. But now, thanks to gene editing techniques like CRISPR, we can also remove the bad genes.

Presumably such a gene swap is what Charlotte did to beget Maisie. Whatever she did, we know that the important part is that she made the change in every single cell of her toddlers body, which is difficult to envision. Several characters say every single cell so we know it is true, although with current technology, that is not possible. Gene therapy on a person targets specific cell types that are involved in the disease.

However Charlotte jettisoned the bad genes out of her kid, Maisie is here, and she holds the secret to how pathologist Henry Wu (BD Wong from Law and Order) can rid the world of the locusts: apply the same technology that Charlotte used to knit a genetic change into all the trillions of her daughters cells.

She changed every cell in your body, Wu reminds Maisie. If I can figure out how, I can change the entire swarm before its too late. Your DNA could change the world! If I could, I could fix a terrible mistake that I made, he laments, referring to the bioengineering of the giant locusts.

A large-scale change that eradicates a species, even if locally, is indeed possible with a technology called a gene drive. I explain it here. Its not a very good idea.

The problem of the locusts is so profound that the old characters from the original Jurassic Park, circa 1993, come aboard. They re-enact some of the old bantering and flirting, nearly verbatim.

Laura Derns Ellie Sattler gee-whizzes and eye-bulges. Shes the paleobotanist who stuck her hands in dino doo in the inaugural film. She helpfully identifies the locusts as a species that died out during the Cretaceous period (145 to 66 million years ago).

Grumpy paleontologist Alan Grant (Sam Neill) is still grumpy, while pontificating mathematician Ian Malcolm (Jeff Goldblum, also of The Fly fame) still is plagued by clichs: ethics of genetic power, we must trust in humanity, unforeseen consequences, and the nonsensical we must transform human consciousness. He works for the evil company or so it seems. Synbios work, he spouts, will cure autoimmune disease, cancer, and Alzheimers.

Ian invites Ellie and Alan to visit Synbio. Upon arrival, Ian slips her a key to the locust lab.

Theyve all aged remarkably well, trim and with great hair and skin. This contradicts the change one thing mantra of rapidly-developing dinosaurs; non-aging humans.

Several boring chase scenes ensue, the one of barbecuing locusts the most intriguing. The main characters spend about 45 minutes running around the Synbio facility to steal a sample of DNA. It is unclear exactly why theyre doing this, because they can easily get DNA from Maisie, and theyre surrounded by the insects, presumably pooping DNA all over the place.

As the movie drags on, more chase scenes happen, in response to various asset containment breaches. The most exciting is a duel between two ferocious dinos: two apex predators in one place! Ellie chants. Oh my!

In yet another scene, as a horrified Claire looks on in a forest, a ginormous dino slurps up deer like my husband eats Trader Joes potato chips.

Our friends, after many near-death experiences, save the day. The surviving humans flee as dinos burn. At an unnamed later time, Dr. Wu waves a magic wand and the locust problem is solved.

The plot holds together loosely. What kept me awake, once Id gotten over the novelty of the dinosaurs (yes, they have feathers), were the scenes borrowed from other films and TV shows.

Owen spies a pterodactyl on the wing of a plane that resembles Star Wars Millennium_Falcon, echoing the Nightmare at 20,000 Feet Twilight Zone episode in which William Shatner (aka Captain Kirk) spies a monster on the wing of a plane.

Why did it have to be snakes? uttered Indiana Jones after plunging into a serpent-filled chasm. Nobody said thered be bugs! is the new version.

Claire bails out of the Millennium Falcon and evokes the winged monkeys of the Wizard of Oz. Later on, shes spies small dinos pop up from the shrubbery like the munchkins along the yellowbrick road.

Claire and Owen sneak into the locust lab wearing white suits that look EXACTLY like the deployed sperm in Woody Allens Everything You Always Wanted to Know About Sex.

For no clear reason, theres a re-enactment of the cantina scene from Star Wars, complete with locusts turning on a barbecue spit, gambling, and drinking.

Despite the absurdity of populating a planet with full-grown, bellowing dinosaurs in a fraction of time, Ill go back for the next installment of Jurassic World. Meanwhile, Id like to know how Ellie, Alan, and Ian aged so well.

Ricki Lewis has a PhD in genetics and is a science writer and author of several human genetics books.She is an adjunct professor for the Alden March Bioethics Institute at Albany Medical College.Follow her at herwebsiteor Twitter@rickilewis

A version of this article was originally posted atPLOSand has been reposted here with permission. PLOS can be found on Twitter@PLOS

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DNA and gene editing are the real stars of the new Jurassic Park movie - Genetic Literacy Project

CRISPR Therapeutics(CRISPR) could be on the cusp of achieving a first in gene therapy, the first company to achieve regulatory approval for a CRISPR-Cas9 program. The company is anticipating the filing of a Biologics License Application for CTX001, a potential cure for transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD).

On Tuesday, the Cambridge, MA-based gene editing company hosted an Innovation Day presentation that highlighted its pipeline and the promise that its multiple approaches have in mitigating or even curing different diseases. Chief Executive Officer Samarth Kulkarni said it has been less than 10 years since Jennifer Doudna and Emmanuelle Charpentier first published an article in the journal Science about gene editing technology and less than eight years since CRISPR Therapeutics was founded on that technological promise. Now, the company is on the cusp of vying for potential approval of its CRISPR therapy, he said.

CTX001, also known as exa-cel, is a CRISPR-Cas9-based gene editing therapy for both TDT and SCD. Earlier this month, CRISPR and its partner Vertex Pharmaceuticals released positive data for exa-cel in TDT and SCD. In the first space, data showed that 42 of 44 patients with TDT who received exa-cel remained transfusion free for up to 37.2 months. The two patients who were not transfusion free had 75% and 89% reductions in transfusion volume, respectively, the companies said. In SCD, all 31 patients with disease characterized by recurrent vaso-occlusive crises (VOCs) were free of the issues following treatment with the gene therapy. Data showed the patients had a duration of up to 32.3 months, CRISPR and Vertex reported. The two companiesexpanded their partnership in this space last year.

We are extremely excited about exa-cel, Kulkarni said.

Phuong Khanh (P.K.) Morrow, CRISPRs new chief medical officer, echoed Kulkarni, saying that she believes exa-cel will be the first CRISPR-Cas9 product approved for both TDT and sickle cell disease. Her prediction comes about a week after an advisory committee with the U.S. Food and Drug Administration recommended approval of a different gene therapy approach for these diseases that were developed by bluebird bio.

In immuno-oncology, CRISPR is also blazing a trail with its approach, particularly with CTX130, a donor-derived gene-edited allogeneic CAR T therapy that targets CD70, which is expressed on various solid tumors and hematologic malignancies. The company is developing the asset for solid tumors, such as renal cell carcinoma, as well as T cell and B cell hematologic cancers. Morrow suggested the positive data the company has seen with CRX130 in both solid and hematologic tumors is something of the Holy Grail that researchers have been searching for."

In May, CRISPR presented exciting CTX130 data from two Phase I studies for relapsed or refractory renal cell carcinoma and various subtypes of lymphoma. The company reported positive early signs, including an overall response rate of 71% from patients with T-cell lymphoma. Of those, 29% experienced a complete response, CRISPR said.

In renal cell carcinoma, cancer that expresses high levels of CD70, CTX130 is also showing significant promise, even leading to a complete response in one patient. Renal cell carcinoma represents a high unmet need, with less than 20% of patients surviving beyond five years. About 40% of RCC patients have shown poor response rates to current therapies.

There is a high potential opportunity with CTX130 because of the CD70 expression in RCC, Morrow said.

Beyond those two assets, CRISPR is also advancing other therapeutics. The company is also developing VCTX210, a potential treatment for type 1 diabetes that is being co-developed with ViaCyte. VCTX210 is an allogeneic, gene-edited, stem cell-derived therapy designed to generate pancreatic cells that can evade recognition by the immune system, which would otherwise destroy them. Earlier this year, the companies dosed the first patient in a Phase I study. As BioSpace previously reported, the goal is for the cell line to be differentiated into pancreatic endoderm cells, generatingglucose-responsive insulin-secreting cells in the patient. It is expected that about 10 patients will be included in the study. Data is anticipated by the end of the year.

While those programs for CRISPR Therapeutics stand out, Kulkarni said the company sees significant potential with its total pipeline, with a chance to delve into multiple disease indications and potentially bring new treatment options to patients.

Were very excited about the potential, he said.

Original post:
CRISPR Therapeutics Eyes Historic First in Gene Therapy - BioSpace

Through the partnership, the CGT Catapult will work with UK academic centers of excellence and the UK DRI to identify new AAV-based gene therapies with high potential to become new medicines for dementia. The organizations will then create detailed development plans for each project and potentially conduct early research activities in order to prepare assets for further investment.

With a team of more than 400 cell and gene therapy experts and headquarters at St Guys hospital in London, the CGT Catapult is an organization dedicated to the advancement of cell and gene therapies.

Meanwhile, the national UK Dementia Research Institute (UK DRI) is the single biggest investment in dementia research in the UK with more than 750 researchers. It was established in 2017 by the Medical Research Council, Alzheimers Society and Alzheimers Research UK and is hosted across six top UK universities: University of Cambridge, Cardiff University, University of Edinburgh, Imperial College London and Kings College London, with its central hub at University College London.

The UK DRIs partnership with the CGT Catapult aims to leverage the expertise and resource of both organizations to accelerate the translation of novel gene therapy approaches to the clinic.

Matthew Durdy, CEO of CGT Catapult, said: Dementia is increasing, under-researched and has very limited treatment options. Cell and gene therapies have in the past shown to be highly effective in treatment areas where other therapies have had limited success. It is therefore vital that we fully explore how cell and gene therapies could be used to address this unmet medical need, and we look forward to working closely with the UK DRI to identify and accelerate the most promising therapies.

Dementia has been one of the leading causes of mortality in the UK since 2015.Over 850,000 people in the UK have dementia and the number of people with dementia will continue to grow as the population ages.

While new and improved treatments for heart disease and cancer are reducing mortality, a lack of effective treatment options for neurodegenerative conditions means that dementia-related deaths continue to rise.

Originally posted here:
New collaboration to accelerate advancements in gene therapies for dementia - BioPharma-Reporter.com

Chronic pain affects millions of people worldwide, yet popular treatments for pain including surgery and opioid medications can have disastrous side effects of their own. But with $1.8 million in funding from the National Institute of Neurological Disorders and Stroke (NINDS), a University of New England researcher will explore non-opioid treatments for chronic pain at the cellular level.

Benjamin Harrison, B.Sc., Ph.D., assistant professor of biochemistry and nutrition, will use the five-year R01 grant from the National Institutes of Health to study how to reduce the excitability of nociceptors, which are neurons that transmit pain signals in response to painful injuries.

Harrison and his team have discovered that nociceptors contain a protein called "CELF4, an RNA binding protein they theorize inhibits the production of pro-nociceptive, or pro-pain-sensing, cellular components. Harrisons research will focus on delivering CELF4 into pain neurons, where this protein will limit the synthesis of ion-channels, receptors, and other molecules that sensitize them.

Specifically, the researchers will study if a locally administered adeno-associated virus can stimulate production of CELF4 and reduce pain in those areas an approach known as gene vector therapy.

Harrison remarked that the innovative approach could prove beneficial for those living with chronic pain but who do not want to undergo surgeries which can be expensive and leave people with no sensation at all or use powerful pain-reducing medications like addictive opioids.

There are some chronic pain conditions that are simply intolerable, and people with those conditions are willing to do severe surgeries to reduce their pain, Harrison remarked. Using this novel gene therapy vector approach, we can develop pain therapies that are less invasive than surgery and carry fewer risks than conventional opioid medications.

Future directions for the research could include partnerships with clinicians for clinical trials, Harrison said.

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UNE researcher awarded $1.8 million to study chronic pain relief through gene therapy - University of New England

DUBLIN, June 20, 2022 /PRNewswire/ -- The "Gene Therapy Global Market Report 2022: By Gene, By Vector, By Application, By End-User" report has been added to ResearchAndMarkets.com's offering.

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The global gene therapy market is expected to grow from $5.77 billion in 2021 to $7.37 billion in 2022 at a compound annual growth rate (CAGR) of 27.8%.

The growth is mainly due to the companies resuming their operations and adapting to the new normal while recovering from the COVID-19 impact, which had earlier led to restrictive containment measures involving social distancing, remote working, and the closure of commercial activities that resulted in operational challenges.

The market is expected to reach $21.25 billion in 2026 at a CAGR of 30.3%

The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs. Gene therapy is used to replace faulty genes or add new genes to cure disease or improve the body's ability to fight disease. Only goods and services traded between entities or sold to end consumers are included.

The main types of gene therapy are antigen, cytokine, suicide gene and others. Antigen is a poison or other foreign substance that triggers an immunological response in the body, including antibody formation. The different vectors include viral vector, non-viral vector, others and is used in oncological disorders, rare diseases, cardiovascular diseases, neurological disorders, infectious diseases, others. it is implemented in various sectors such as hospitals, homecare, specialty clinics, others.

Incidences of cancer and other target diseases have been increasing significantly, which is calling for effective treatments, driving the growth of the gene therapy market. The rise in the number of cancer cases across the globe is likely to contribute to the growth of the gene therapy market during the forecast period. According to the American Cancer Society, there were 1.7 million new cases and 0.6 million cancer deaths in 2019 in the USA.

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The four most common types of cancer worldwide are lung, prostate, bowel, and female breast cancer, accounting for 43% of all the new cancer cases. Therefore, the rise in the cancer incidence rate globally is anticipated to boost the demand for the gene therapy market over the forthcoming years. Gene therapy is one of the most effective treatments in oncology.

In this treatment, new genes are introduced into a cancerous cell or the surrounding tissue to cause cell death or slow the growth of cancer. For instance, in September 2019, RMIT University, Australia has found that non-viral gene therapy can be used to speed up cancer research, which can bring patient-friendly cancer treatment in the market.

The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for cancer and other diseases has influenced the health conditions of the population and has led to a low average life expectancy.

Luxturna, a one-time treatment for acquired retinal eye disease, costs $850,000 in the US and 613,410 in the UK, despite a markdown that is applied through Britain's National Health Service. Zolgensma, for spinal muscular atrophy, is valued at $2.1 million in the US and Zynteglo, which focuses on a rare genetic blood disorder, costs $1.78 million, thus restraining the growth of the market.

The use of machine learning and artificial intelligence is gradually gaining popularity in the gene therapy market. Artificial intelligence (AI) is the simulation of human intelligence in machines, which are programmed to display their natural intelligence. Machine learning is a part of AI.

Machine learning and AI help companies in the gene therapy market to conduct a detailed analysis of all relevant data, provide insights between tumor and immune cell interactions, and offer a more accurate evaluation of tissue samples often conflicted between different evaluators. It is also expected to reduce turnaround time and also the cost of gene therapies.

Major players in the gene therapy market are

Novartis AG

Bluebird Bio, inc.

Spark Therapeutics, inc.

Audentes Therapeutics

Voyager Therapeutics

Applied Genetic Technologies Corporation

UniQure N.V.

Celgene Corporation

Cellectis S.A.

Sangamo Therapeutics

Gilead Lifesciences, inc.

Orchard Therapeutics

Sibiono GeneTech Co., Ltd.

Gensight Biologics S.A.

Shanghai Sunway Biotech Co., Ltd.

Biogen

Sarepta Therapeutics, inc.

Sangamo Therapeutics

Audentes Therapeutics

Regenxbio, inc.

Gegenxbio, inc.

Oxford BioMedica plc.

Dimension Therapeutics, inc.

Bristol-Myers Suibb Company

Sanofi

Taxus Cardium Pharmaceuticals Group, inc. (Gene Biotherapeutics)

Shrine plc.

Benitech Biopharma

Transgene

Epeius Biotechnologies Corp.

Calimmune, inc.

Key Topics Covered:

1. Executive Summary

2. Gene Therapy Market Characteristics

3. Gene Therapy Market Trends And Strategies

4. Impact Of COVID-19 On Gene Therapy

5. Gene Therapy Market Size And Growth5.1. Global Gene Therapy Historic Market, 2016-2021, $ Billion 5.1.1. Drivers Of The Market 5.1.2. Restraints On The Market 5.2. Global Gene Therapy Forecast Market, 2021-2026F, 2031F, $ Billion 5.2.1. Drivers Of The Market 5.2.2. Restraints On the Market

6. Gene Therapy Market Segmentation6.1. Global Gene Therapy Market, Segmentation By Gene Type, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

Antigen

Cytokine

Suicide Gene

Others

6.2. Global Gene Therapy Market, Segmentation By Vector, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

Viral Vector

Non-Viral Vector

Others

6.3. Global Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

Oncological Disorders

Rare Diseases

Cardiovascular Diseases

Neurological Disorders

Infectious Diseases

Others

6.4. Global Gene Therapy Market, Segmentation By End Users, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

Hospitals

Homecare

Specialty Clinics

7. Gene Therapy Market Regional And Country Analysis7.1. Global Gene Therapy Market, Split By Region, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion 7.2. Global Gene Therapy Market, Split By Country, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

For more information about this report visit https://www.researchandmarkets.com/r/wzvr47

Media Contact:

Research and MarketsLaura Wood, Senior Managerpress@researchandmarkets.com

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Global Gene Therapy Market Research Report 2022-2031 Featuring Major Players - Novartis AG; Bluebird bio, Inc.; Spark Therapeutics, Inc; Audentes...

Whether it's due to jetpacks, augmented reality, or gene therapy, the future of mountaineering might look very different. In terms of gene therapy, this technology may have the potential to make the mountains more accessible and much safer.

Through years ofresearch into a sometimes fatal condition commonly known as 'altitude sickness,' scientists have determined that symptoms of the condition, also called chronic mountain sickness (CMS), are most likely the result of a population-specific "maladaptation" that impactsup to half of the human population. In other words, some groups of people are genetically inclined to experience severe altitude sickness symptoms because their genes limit their ability to to adjust to high elevation environments. The basis for this assumption lies in how some populations that have traditionally lived at higher elevations around the globe, like those in the Himalayas, tend to be less susceptible to the condition.

If altitude sickness is indeed the result of genetics developed over time, this may mean that the condition could be treated with gene therapy. Research has already found several "candidate genes" that could be the underlying cause behind the maladaptation, meaning that if these genes were successfully targeted with treatment, it may help to limit effects of the condition.

As might be expected, gene therapy is very complicated. Cleveland Clinicdescribes the process as a doctor delivering a healthy copy of a gene to cells inside the body via injection or IV with the hope that the healthy gene will "replace a damaged (mutated) gene, inactivate a mutated gene, or introduce an entirely new gene." Obviously, a ton of research and development must also go into creating that healthy gene first, but this could mean that if scientists are able to fully determine what aspect of the genetic code is causing altitude sickness, they may be able to alter it in a way that eliminates the maladaptation.

If gene therapy technology as it relates to altitude sickness continues to develop, it may add another tool that some people could use to better their mountain climbing potential. Having another option for combating altitude sickness would be hugely beneficial, as only a couple medicines are available for preventing altitude sickness (dexamethasone and acetazolamide,2021) and only one medicine is recommended as a truly effective treatment (dexamethasone).

Not only would a gene therapy option make exploring the mountains safer for many of those already doing so, it would also make high-elevation landscapes more accessible for those that may avoid them due to CMS concerns. While many people don't start to feel the effects of elevation until they've spent several hours above 8,000 feet, others can experience symptoms of a devastating degree at a much lower elevation in a much shorter time frame during a layover at Denver International Airport, for example. For those that face altitude sickness concerns on a regular basis, this technology could be life-changing, and for the rest of us, it could make the mountains a lot more fun.

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Here's one way gene therapy could change the future of mountain climbing - Out There Colorado

The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20222031.

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

The following factors are likely to contribute to the growth of the Europe cell and gene therapy market during the forecast period:

CMOs Offering Vector Manufacturing Services for Cell and Gene Therapy Companies Robust Cell & Gene Therapies in the Pipeline Increase in Strategic Acquisitions Regulatory Support for Cell and Gene Therapy Products

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The study considers the present scenario of the Europe cell and gene therapy market and its market dynamics for the period 20222031. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Europe Cell and Gene Therapy Market Segmentation

The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patients quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

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INSIGHTS BY GEOGRAPHY

Germany, France, the UK, Italy, and Spain play a significant role in the Europe cell and gene therapy market. Clinical trials and the number of manufacturing facilities are increasing slowly in the European region. The region has become a major R&D destination for several vendors as the funding for cell & gene therapies is increasing. Europe has supported collaborative efforts in gene transfer and gene therapy research. In addition, the target patient population is increasing across Europe; there were an estimated 3.9 million new cases of cancer and 1.9 million cancer deaths in Europe in 2018. In addition, the prevalence surveys in the UK and Denmark indicate that there are 34 people with one or more wounds per 1,000 people. Favorable government support in terms of product approvals, reimbursement and coverage, and high R&D funding to academic institutes that are involved in the development of cell and gene therapies are expected to boosting the market in Europe.

INSIGHTS BY VENDORS

Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

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Europe Cell and Gene Therapy Market is Poised to Grow at a CAGR of 23% during the Forecast Period of 2022-2031 - Digital Journal

OAKDALE, Minn., June 21, 2022 /PRNewswire/ -- BlueAllele Corporation, a biotechnology company focused on advancing transformational gene correction technologies, announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 11,365,407 (the '407 Patent) related to its proprietary in vivo gene correction methodologies.

BlueAllele was founded with the ambitious objective to develop methodologies for single-dose treatments to cure the most debilitating and devastating genetic diseases. Over 5,000 genetic diseases, which are caused by mutations within a gene, have been identified and diagnosed in humans. The mutations can inactivate gene function, referred to as loss-of-function mutations, or create an undesirable function, referred to as gain-of-function mutations. BlueAllele has developed transformational gene correction technologies utilizing its proprietary PALIDON repair template platforms to achieve gene correction that is precise, durable, and capable of treating a broad range of both loss-of-function and gain-of-function mutations.

Dr. Nicholas Baltes, BlueAllele's chief scientific officer, said, "We saw that there was a large gap in the capabilities of the genetic tools for gene correction and the development of effective, safe and lifelong therapies. We set out to close the gap."

Unlike traditional gene therapy where the corrective sequences remain extrachromosomal, PALIDON is integrated into the cell's DNA thereby providing potential lifelong therapeutic effects. Further, unlike gene knockout approaches where gene function is destroyed, PALIDON provides replacement sequence for restoration of gene function.

The distinctive palindromic design of BlueAllele's PALIDON repair templates has unlocked the full potential of the cell's highly active NHEJ pathway. In doing so, PALIDON can increase the efficacy of gene correction events, improve safety and be used for the therapeutic treatment of a wide range of diseases. BlueAllele has expanded upon this foundational feature with the design of additional repair template platforms for treating specific classes of genetic diseases. PALIDON+ was designed for autosomal dominant diseases and functions to remove unwanted gene products while simultaneously restoring normal protein function. PALIDON DT was designed to address repeat expansion diseases and functions to prevent transcription through the expansion.

"We are excited about the potential of our PALIDON platforms for gene correction in patients," said Joseph B. Saluri, BlueAllele's chief executive officer. "What was once considered beyond reach is now closer to becoming a reality."

About BlueAllele's Patents

The newly granted '407 Patent includes claims covering adeno-associated viral vectorsharboring BlueAllele's proprietary PALIDON transgene design. The claims also cover adeno-associated viral vectors harboring a PALIDON transgene containingFactor 9 coding sequences for treating patients with hemophilia B.

U.S. Patent 11,254,930 (the '930 Patent) includes claims covering recombinant nucleic acids containing BlueAllele's novel PALIDON transgene structure, which includes splice acceptors, coding sequences and terminators for correcting gene function. The PALIDON transgenes can be used with any nuclease, any delivery system and any application for treating a genetic disease in any tissue or cell.

U.S. patent 11,091,756 (the '756 Patent) includes claims covering methods for using PALIDON to edit genes in any cell type and system, in vitro or in vivo applications, as well as its use for correcting any gene in the genome.

The '407, '930 and '756 Patents are exclusively owned by BlueAllele Corporation and provide broad coverage for the development and advancement of human gene correction technologies.

BlueAllele Corporation is a biotechnology company committed to advancing transformational gene correction technologies, including its patented PALIDON PALIDON+ and PALIDON DT repair template platforms. BlueAllele is a member of Medical Alley, The Global Epicenter of Health Innovation and Care. For more information, visit http://www.blueallele.com.

BlueAllele is seeking strategic collaborators for leading innovation for gene correction technologies. If interested, please contact us via http://www.blueallele.com/contact.

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BlueAllele Corporation continues to advance therapeutic approaches using novel gene correction technologies and secures third patent from the USPTO -...

The recently released report on the global Gene Therapy for Ovarian Cancer Market 2022 to 2029 provides a comprehensive evaluation of the respective industry that contains several aspects of product specifications, Gene Therapy for Ovarian Cancer industry segmentation supported by numerous characteristics, and an analysis of the competitors landscape. The new research document assesses the desired prospects and existing industry position, offering powerful insights and crucial updates on the corresponding segments included in the global Gene Therapy for Ovarian Cancer market for the projected period from 2022 to 2029.

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Takara BioVBL TherapeuticsCELSIONTargovax

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IntravenousIntratumoralIntraperitoneal

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Ovarian Cancer (unspecified)Recurrent Ovarian Epithelial CancerPlatinum-Resistant Ovarian Cancer

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North America Market(United States, Canada, North American country and Mexico),Europe Market (Germany, Gene Therapy for Ovarian Cancer France, UK, Russia and Italy),Asia-Pacific Market (China, Gene Therapy for Ovarian Cancer Japan and Korea, Asian nation, India and Southeast Asia),South America Market (Brazil, Argentina, Republic of Colombia etc.),Middle East & Africa Market (Saudi Arabian Peninsula, UAE, Egypt, Nigeria and South Africa)

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Global Gene Therapy for Ovarian Cancer Market Prescriptive Research, Production Information 2022-2029 |Takara Bio, VBL Therapeutics, CELSION, Targovax...

CAMBRIDGE, Mass., June 15, 2022 (GLOBE NEWSWIRE) -- BiogenInc.(Nasdaq: BIIB) will present new data from clinical studies aimed at assessing remaining unmet needs for people living with spinal muscular atrophy (SMA) and evaluating the potential impact of SPINRAZA (nusinersen) in different patient populations at the SMA Research & Clinical Care Meeting hosted by Cure SMA this week in Anaheim, Calif. Biogen is a presenting sponsor of Cure SMAs 2022 Annual SMA Conference, the worlds largest meeting dedicated to SMA research and care.

The data we are presenting at this years Cure SMA conference including the latest updates from the RESPOND and DEVOTE studies reinforce Biogens commitment to evaluating the potential of SPINRAZA to further improve clinical outcomes for individuals with SMA, said Maha Radhakrishnan, M.D., Chief Medical Officer at Biogen. There are key unmet needs within the SMA community and we are committed to addressing these through our ongoing research that includes active enrollment in three global clinical studies.

SMA Research Updates Growing enrollment in the RESPOND study indicate there are residual unmet clinical needs in infants and toddlers with SMA following treatment with Zolgensma (onasemnogene abeparvovec). The Phase 4 study is evaluating the clinical benefit and safety of SPINRAZA in infants and toddlers with SMA who have unmet needs following treatment with the gene therapy.

Since initial findings from nine patients were shared in March 2022, baseline and safety data from 16 patients enrolled in RESPOND (as of November 2021) are being presented. All enrolled study participants reported suboptimal clinical status across a variety of measures at baseline, with 13 of 16 showing this in multiple areas, including motor and respiratory functions and swallowing/feeding ability. After beginning SPINRAZA treatment, initial safety findings (median duration of 132.5 days) show three participants experienced a serious adverse event (AE) during the study period; none of these events were considered related to SPINRAZA treatment. The RESPOND study (NCT04488133) is currently enrolling participants at 20 sites worldwide; more information about the eligibility criteria is available at clinicaltrials.gov.

Biogen will also share final data from Part A of the ongoing, three-part DEVOTE study evaluating the safety and tolerability of investigational, higher doses of nusinersen.* Results from Part A, an open-label safety evaluation period in children and teens with later-onset SMA, suggest that a higher dosing regimen (28 mg) of nusinersen leads to higher levels of the drug in the cerebrospinal fluid and is generally well-tolerated, with most AEs reported considered to be mild in severity. The most common AEs reported were headache and procedural pain. Two serious AEs (fall, femur fracture) were reported in one participant during the study period. No AEs were considered related to nusinersen and some were related to treatment administration. The totality of Part A data supports further development of a higher dose ofnusinersen.

Currently, Part B and Part C of DEVOTE evaluating an investigational, higher dose of nusinersen are enrolling at 52 sites worldwide. Information on the DEVOTE trial (NCT04089566) is available at clinicaltrials.gov.

Featured SPINRAZA Data Presentations Include:

*Nusinersen is currently commercialized under the brand name SPINRAZA and the U.S. Food and Drug Administration-approved dose is 12 mg. As a foundation of care in SMA, more than 13,000 individuals have been treated with SPINRAZA worldwide.1

About SPINRAZA(nusinersen)The SPINRAZA clinical development program encompasses 10 clinical studies, which have included more than 300 individuals across a broad spectrum of patient populations,2including two randomized controlled studies (ENDEAR and CHERISH). The ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in antisense therapeutics. Please click here forImportant Safety Informationandfull Prescribing Informationfor SPINRAZA in the U.S., or visit your respective countrys product website.

About Spinal Muscular Atrophy (SMA)SMA is a rare, genetic, neuromuscular disease that affects individuals of all ages. It is characterized by a loss of motor neurons in the spinal cord and lower brain stem, resulting in progressive muscle atrophy and weakness.3SMA is caused by a deficiency in the production of survival motor neuron (SMN) protein due to a damaged or missingSMN1gene, with a spectrum of disease severity.3Some individuals with SMA may never sit; some sit but never walk; and some walk but may lose that ability over time.4In the absence of treatment, children with the most severe form of SMA would not be expected to reach their second birthday.3

SMA impacts approximately 1 in 10,000 live births,5-8is a leading cause of genetic death among infants9 and causes a range of disability in teenagers and adults.4

About BiogenAs pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the worlds first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first and only approved treatment to address a defining pathology of Alzheimers disease. Biogen is also commercializing biosimilars and focusing on advancing one of the industrys most diversified pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need.

In 2020, Biogen launched a bold 20-year, $250 million initiative to address the deeply interrelated issues of climate, health, and equity. Healthy Climate, Healthy Lives aims to eliminate fossil fuels across the companys operations, build collaborations with renowned institutions to advance the science to improve human health outcomes, and support underserved communities.

We routinely post information that may be important to investors on our website atwww.biogen.com.Follow us on social media-Twitter,LinkedIn,Facebook,YouTube.

Biogen Safe HarborThis news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, about the potential benefits, safety and efficacy of nusinersen; the results of certain real-world data; our research and development program for the identification and treatment of SMA; clinical development programs, clinical trials and data readouts and presentations; the potential benefits and results from treatment of SMA; and risks and uncertainties associated with drug development and commercialization. These statements may be identified by words such as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, plan, possible, potential, will, would and other words and terms of similar meaning. You should not place undue reliance on these statements or the scientific data presented.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation risks relating to the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis, including from the DEVOTE, RESPOND and ASCEND studies; the risk that we may not fully enroll our clinical trials, or enrollment will take longer than expected; failure to obtain regulatory approvals in other jurisdictions; risks of unexpected costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies; product liability claims; third party collaboration risks; and the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

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New Data Presented at Cure SMA Reveal Residual Unmet Needs in Young SMA Patients Treated With Gene Therapy and Suggest Further Potential of Using...