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

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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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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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Significant Players involved in the Gene Therapy for Ovarian Cancer market report:

Takara BioVBL TherapeuticsCELSIONTargovax

Gene Therapy for Ovarian Cancer Market splits into Product Types:

IntravenousIntratumoralIntraperitoneal

Key Applications of the Gene Therapy for Ovarian Cancer market are:

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

The Geographical assessment of the Gene Therapy for Ovarian Cancer market is:

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)

The new study report gives a systematic and descriptive approach to the global Gene Therapy for Ovarian Cancer market. It constructs industry dynamics, the overall extent of growth in distinct segments, regions as well as various other parameters that have been immensely effective in enlarging the industry size and value. Therefore, this document aimed at delivering a clear perspective of all possible conditions and structures in the global Gene Therapy for Ovarian Cancer market.

Read Complete Analysis Report for Better Understanding (Description, TOC, List of Tables & Figures and Many More): https://marketresearchexpertz.com/report/global-gene-therapy-ovarian-cancer-market-461384

Vital Questions Answered in the Gene Therapy for Ovarian Cancer market report:

What is the projected growth rate, share, and size of the Gene Therapy for Ovarian Cancer market? What are the available driving forces in the Gene Therapy for Ovarian Cancer market for the estimated period from 2022 to 2030? What was the value of the Gene Therapy for Ovarian Cancer market in Europe, North America & Asia Pacific? What is the forecasted industry analysis of the global Gene Therapy for Ovarian Cancer market? Who are the prominent industry players of the Gene Therapy for Ovarian Cancer market globally? How industry players have grabbed a competitive edge over other competitors? At what CAGR is the Gene Therapy for Ovarian Cancer market expected to expand? What are the crucial challenges and threats limiting the progress of the Gene Therapy for Ovarian Cancer industry? What are the topmost trends in the Gene Therapy for Ovarian Cancer market?

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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...

SOLANA BEACH, Calif., May 20, 2022 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. ( CLPT) (the Company), a global therapy-enabling platform company providing navigation and delivery to the brain, today congratulates partner PTC Therapeutics for receiving a positive opinion from the European Medicines Agencys (EMA) Committee for Medicinal Products for Human Use (CHMP) recommending its gene therapy treatment Upstaza (eladocagene exuparvovec), formerly PTC-AADC, for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The use of ClearPoint Neuros proprietary CE Marked SmartFlow Neuro Ventricular Cannula for minimally invasive infusion of the gene therapy is included in the submission for administration of Upstaza.

This is a significant milestone for PTC Therapeutics, for ClearPoint and for biologics researchers everywhere as it marks the very first positive CHMP recommendation for direct injection to the brain of a gene therapy to treat a severe, highly morbid and fatal neurological disorder, commented Joe Burnett, President and CEO of ClearPoint Neuro. This milestone is incredibly important for a couple of reasons. First, it provides a potential path for other therapies to follow and sets an important precedent for regulatory approval of direct administered gene therapies to the brain. And second, we see this as a validation of ClearPoints biologics partnership strategy and our rigorous bench, preclinical and clinical experience. We believe this announcement will give our 45 existing and future pharmaceutical, academic and biotech partners the confidence that SmartFlow is able to pass through the necessary regulatory scrutiny and can de-risk the commercialization efforts of many different drugs to multiple targets in the brain.

About aromatic L-amino acid decarboxylase (AADC) deficiency

AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life physical, mental, and behavioral. The suffering of children with AADC deficiency may be exacerbated by episodes of distressing seizure-like oculogyric crises, which can happen daily and last for hours, causing the eyes to roll up in the head, frequent vomiting, behavioral problems, difficulty sleeping, and life-threatening complications such as respiratory infections and gastrointestinal problems.

There is no disease-modifying treatment approved for AADC deficiency, and the lives of affected children are severely impacted, and shortened, with the use of many different medications to help manage symptoms. Ongoing physical, occupational and speech therapy, and interventions, including surgery, to manage potentially life-threatening complications such as infections, severe feeding, and breathing problems also are often required.

About the SmartFlow Cannula

With over 5,000 cannulas sold to date, SmartFlow is the only co-labeled device to gain approval by a regulatory agency for delivery of an approved gene therapy to the brain. The industry-leading cannula is used by many of ClearPoint Neuros 45 pharmaceutical, academic, and biotech partners to bypass the blood brain barrier and deliver therapeutics to regions of interest using Convection Enhanced Delivery (CED) under direct image guidance. The SmartFlow cannula has received 510(k) clearance from the FDA for use in the United States for the aspiration of cerebrospinal fluid or injection of the chemotherapy drug Cytarabine into the ventricles. It has also been CE marked to deliver approved fluids into the brain or aspiration of CSF. SmartFlow is being utilized in approved clinical and preclinical studies for various research and drug trials. This device is not intended for implant and is intended for single patient use only.

About ClearPoint Neuro

ClearPoint Neuros mission is to improve and restore quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of the Companys current product portfolio include deep brain stimulation, laser ablation, biopsy, neuro-aspiration, and delivery of drugs, biologics, and gene therapy to the brain. The ClearPoint Neuro Navigation System has FDA clearance, is CE-marked, and is installed in over 60 active sites in the United States, Canada, and Europe. ClearPoint Neuro is partnered with approximately 45 biologics/pharmaceutical companies and academic centers, providing solutions for direct CNS delivery of therapeutics in preclinical studies and clinical trials worldwide. To date, more than 5,000 cases have been performed and supported by the Companys field-based clinical specialist team, which offers support and services to our customers and partners worldwide. For more information, please visit http://www.clearpointneuro.com.

Forward-Looking Statements

Statements herein concerning the Companys plans, growth and strategies may include forward-looking statements within the context of the federal securities laws. Statements regarding the Company's future events, developments and future performance, as well as management's expectations, beliefs, plans, estimates or projections relating to the future, are forward-looking statements within the meaning of these laws. Uncertainties and risks may cause the Company's actual results to differ materially from those expressed in or implied by forward-looking statements. Particular uncertainties and risks include those relating to: the impact of the COVID-19 pandemic and the measures adopted to contain its spread; future revenue from sales of the Companys ClearPoint Neuro Navigation System products; the Companys ability to market, commercialize and achieve broader market acceptance for the Companys ClearPoint Neuro Navigation System products; and risks inherent in the research and development of new products. More detailed information on these and additional factors that could affect the Companys actual results are described in the Risk Factors section of the Companys Annual Report on Form 10-K for the year ended December 31, 2021, and the Companys Quarterly Report on Form 10-Q for the three months ended March 31, 2022, both of which have been filed with the Securities and Exchange Commission.

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ClearPoint Neuro Congratulates Partner PTC Therapeutics on Receiving Positive CHMP Opinion for Gene Therapy to Treat AADC Deficiency - GuruFocus.com

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a biotechnology company with a mission to discover, develop and deliver curative therapies that address the underlying causes of heart disease, today announced that it has completed the build-out and operational launch of its Genetic Medicines Manufacturing Center in Union City, California. Tenaya is advancing a pipeline of therapeutic candidates, including several adeno-associated virus (AAV) gene therapies, for the potential treatment of both rare and prevalent forms of heart disease.

Tenaya made an early, strategic commitment to internalize several core capabilities to optimize the safety, efficacy, and supply of our product candidates on behalf of patients. With todays announcement we have made a big leap forward on that commitment by establishing end-to-end in-house manufacturing capabilities for our pipeline of AAV-based gene therapies, said Faraz Ali, Chief Executive Officer of Tenaya. The operational launch of Tenayas Genetic Medicines Manufacturing Center represents an important milestone as we prepare to advance our robust pipeline of potentially first-in-class cardiovascular therapies into initial clinical studies.

Tenayas Genetic Medicines Manufacturing Center is designed to meet regulatory requirements for production of AAV gene therapies from discovery through commercialization under Current Good Manufacturing Practice (cGMP) standards. Initial production efforts will support first-in-human studies of Tenayas lead gene therapy, TN-201. TN-201 is being developed for the treatment of genetic hypertrophic cardiomyopathy (HCM) due to MYBPC3 gene mutations. Tenaya plans to submit an Investigational New Drug (IND) application for TN-201 to the U.S. Food and Drug Administration (FDA) in the second half of this year. The facility will also support cGMP production for TN-401, Tenayas gene therapy program being developed for the treatment of genetic arrhythmogenic right ventricular cardiomyopathy (ARVC) due to PKP2 gene mutations, for which the company plans to submit an IND to the FDA in 2023.

The investment in our own world-class manufacturing facility provides Tenaya with greater control over product attributes, quality, production timelines and costs, which we believe will ultimately translate into better treatments for patients, said Kee-Hong Kim, Ph.D., Chief Technology Officer of Tenaya Therapeutics. Tenayas Genetic Medicines Manufacturing Center complements our established internal genetic engineering and drug discovery capabilities and is designed to meet our near- and long-term needs such that we can readily scale and expand as our pipeline matures and evolves.

Tenaya completed customization of approximately half of the 94,000 square foot facility to incorporate manufacturing suites and labs, office space and storage. Utilizing a modular design, the state-of-the-art facility is now fully operational with initial capacity to produce AAV-based gene therapies at the 1000L scale, utilizing Tenayas proprietary baculovirus-based production platform and suspension Sf9 cell culture system. The excess space and modular design of the Genetic Medicines Manufacturing Center is intended to provide Tenaya with considerable flexibility to expand manufacturing capacity by increasing both the number and the scale of bioreactors to meet future clinical and commercial production needs.

The Union City location, approximately 30 miles from Tenayas South San Francisco headquarters, is expected to enable the seamless transition of Tenayas science from early research through commercial manufacturing. The selection of this location is intended to foster a culture of close collaboration across teams at all stages of developing and testing novel AAV capsids, de-risk the translation from research to process development and create opportunities for improvements in production processes. The Genetic Medicines Manufacturing Center is staffed by a growing in-house team with expertise in all aspects of gene therapy manufacture, including process development, analytical development, quality assurance and quality control.

About Tenaya Therapeutics

Tenaya Therapeutics is a biotechnology company committed to a bold mission: to discover, develop and deliver curative therapies that address the underlying drivers of heart disease. Founded by leading cardiovascular scientists from Gladstone Institutes and the University of Texas Southwestern Medical Center, Tenaya is developing therapies for rare genetic cardiovascular disorders, as well as for more prevalent heart conditions, through three distinct but interrelated product platforms: Gene Therapy, Cellular Regeneration and Precision Medicine. For more information, visit http://www.tenayatherapeutics.com.

Forward Looking Statements

This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as potential, will, plans, believe, expected, and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, statements regarding the therapeutic potential of Tenayas pipeline of therapeutic candidates; Tenayas plan to use the cGMP manufacturing facility for the production of TN-201 and TN-401; Tenayas belief that its cGMP manufacturing facility will enable seamless transition from early research through commercial manufacturing and translate into better treatments for patients; the expected timing for submission of IND applications for TN-201 and TN-401; and statements by Tenayas chief executive officer and chief technology officer. The forward-looking statements contained herein are based upon Tenayas current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; Tenayas ability to successfully manufacture product candidates in a timely and sufficient manner that is compliant with regulatory requirements; Tenayas ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; the timing, progress and results of preclinical studies for TN-201, TN-401 and Tenayas other programs; Tenayas ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Tenayas manufacturing and operations, including preclinical studies and planned clinical trials; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Tenayas manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenayas reliance on third parties; Tenayas ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled Risk Factors in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

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Tenaya Therapeutics Launches Operations of New Genetic Medicines Manufacturing Center to Support the Development of Potentially First-In-Class...