First therapy recommended for full marketing authorization in the EU for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants

One-time treatment with Libmeldy has been shown to preserve cognitive and motor function in most patients

Libmeldy is backed by data across 35 patients with follow-up of up to 8 years post-treatment, demonstrating the potential durability of HSC gene therapy

BOSTON and LONDON, Oct. 16, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A, or ARSA, gene), an investigational gene therapy for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.

The CHMPs positive opinion will now be reviewed by theEuropean Commission(EC), which has the authority to grant marketing authorization for Libmeldy in theEuropean Union(EU). A final decision by the EC for Libmeldy is anticipated before the end of 2020. If approved, Libmeldy would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.

MLD is a very rare, severe genetic condition caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them. A majority of these patients pass away in childhood, with palliative care often as their only option.

Todays positive CHMP opinion for marketing authorization of Libmeldy is a remarkable achievement that we share with the MLD community, as it brings us closer to delivering a one-time, potentially transformative therapy for eligible children suffering from this devastating disease, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. Data from the Libmeldy clinical program have demonstrated the potential for long-term positive effects on cognitive development and maintenance of motor function, translating to individual preservation of motor milestones such as the ability to sit, stand and/or walk without support, as well as attainment of cognitive skills like social interactions and school attendance, at ages at which untreated patients show severe motor and cognitive impairments.

Libmeldy is designed as a one-time gene therapy, developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, in which the patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying genetic condition with a single treatment.

This is an important milestone toward making the availability of HSC gene therapy a reality for more patients, and it also is extremely rewarding for our multi-disciplinary team at SR-Tiget who has worked relentlessly along this 15-year journey to move the seminal proof of principle studies to the first in-human testing of this therapy, said SR-Tiget director Luigi Naldini, M.D, Ph.D. The robust and durable clinical benefits observed in early-onset MLD patients who received HSC gene therapy are compelling, especially when compared to the natural history of the disease. These results also further illustrate our view that the HSC gene therapy approach has the potential to deliver transformative effects in other storage diseases as well, especially when the cells are designed to overexpress the functional enzyme and provide an enhanced supply of it to the affected tissues.

As a parent, watching your child start down a seemingly normal developmental path only to suddenly and rapidly lose some or all of his or her abilities is heart-wrenching, and the agony is even more acute knowing no approved therapies currently exist for MLD, said Georgina Morton, Chair of ArchAngel MLD Trust. Todays decision to advance Libmeldy to the final EC approval stage represents a huge step forward for the parents of these young children and for all of us in the MLD community.

We are extremely appreciative of the EMAs expedited and thorough review of Libmeldys marketing authorization application, considering the severity of MLD coupled with the limited treatment options available today for young patients, said Anne Dupraz, chief regulatory officer, Orchard Therapeutics. The Agencys collaboration on this assessment is a testament to their broader public health commitment to ensure timely evaluation of new medicines for diseases where a pressing unmet need exists.

Data Supporting the Clinical Profile of Libmeldy

The positive CHMP opinion is supported by clinical studies of Libmeldy in both pre- and early- symptomatic, early-onset MLD patients. Early-onset MLD encompasses the disease variants traditionally referred to as late infantile (LI) and early juvenile (EJ).

Clinical efficacy was based on the integrated analysis of results from 29 patients with early-onset MLD who were all treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation:

Clinical safety was evaluated in 35 patients with early-onset MLD:

Co-primary endpointsThe co-primary endpoints of the integrated efficacy analysis were Gross Motor Function Measure (GMFM) total score and ARSA activity, both evaluated at 2 years post-treatment. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Pre-symptomatic LI and EJ patients treated with Libmeldy experienced significantly less deterioration in motor function at 2 years and 3 years post-treatment, as measured by GMFM total score, compared to age and disease subtype-matched untreated patients (p0.008). The mean difference between treated pre-symptomatic LI patients and age-matched untreated LI patients was 71.0% at year 2 and 79.8% at year 3. Similarly, the mean difference between treated pre-symptomatic EJ patients and age-matched untreated EJ patients was 52.4% at year 2 and 74.9% at year 3. Although not statistically significant, a clear difference in GMFM total score was also noted between treated early-symptomatic EJ patients and age-matched untreated EJ patients (28.7% at year 2; p=0.350 and 43.9% at year 3; p=0.054).

A statistically significant increase in ARSA activity in peripheral blood mononuclear cells was observed at 2 years post-treatment compared to pre-treatment in both pre-symptomatic patients (20.0-fold increase; p<0.001) and early-symptomatic patients (4.2-fold increase; p=0.004).

At the time of the integrated data analysis, all treated LI patients were alive with a follow-up post-treatment up to 7.5 years and 10 out of 13 treated EJ patients were alive with a follow-up post-treatment of up to 6.5 years. No treatment-related mortality has been reported in patients treated with Libmeldy.

Key secondary endpointsFor EJ patients who were early-symptomatic when treated with Libmeldy, meaningful effects on motor development were demonstrated when these patients were treated before entering the rapidly progressive phase of the disease (IQ85 and Gross Motor Function Classification (GMFC)1). By 4 years post-disease onset, an estimated 62.5% of treated, early-symptomatic EJ MLD patients survived and maintained locomotion and ability to sit without support compared with 26.3% of untreated early-symptomatic EJ MLD patients, representing a delay in disease progression following treatment with Libmeldy.

A secondary efficacy endpoint that measured cognitive and language abilities as quantified by Intelligence Quotient/Development Quotient (IQ/DQ) found:

Clinical safetySafety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in 5 out of 35 patients. Antibody titers in all 5 patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. Treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

About MLD and Investigational Libmeldy

Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in thearylsulfatase-A(ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. Currently, there are no approved treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), formerly OTL-200, is being studied for the treatment of MLD in certain patients. Libmeldy was acquired from GSK inApril 2018and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their jointSan Raffaele-Telethon Institute for Gene TherapyinMilan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

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

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the regulatory approval and commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that our marketing authorization application submitted for Libmeldy may not be approved by the European Commission when expected, or at all; the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US and EU or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, if approved, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended June 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

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

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

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Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire

The girl died at home several months after receiving the therapy (Representational)

A five-year-old girl with a rare neurodegenerative disease died in the US while taking part in a gene therapy trial run by French biotechnology company Lysogene, the firm said Thursday.

The little girl was suffering from Sanfilippo syndrome or mucopolysaccharidosis type III, a rare genetic disease that alters brain development after birth and leads to premature death.

In a statement Lysogene, a company developing gene therapy for central nervous system diseases in children, said "the immediate cause of death is currently unknown" and that there was as yet "no evidence that the event is linked to the study drug administration".

It said it was "profoundly saddened by the passing of this child" and was collecting "additional information" about the circumstances.

The share price of the company dropped 19 percent to 2.05 euros in morning trading in Paris.

The girl was one of 19 people being treated in the trial conducted at eight hospitals in Europe and the United States.

She died at home several months after receiving the therapy, consisting of a single injection, at one of four treatment sites in the US, Lysogene told AFP.

In its statement the company said it was following the remaining 18 patients closely and remained "committed to the LYS-SAF302 development program".

On June 5, the US Food and Drug Administration (FDA) ordered a clinical hold on the trial after observing "localized findings on MRI images at the intracerebral injection sites" suggesting "a potential connection to delivery".

In a statement at the time the company said "no clinical symptoms have been observed that could be directly attributed to the observed MRI findings".

(Except for the headline, this story has not been edited by NDTV staff and is published from a syndicated feed.)

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5-Year-Old Girl, Being Treated In French Gene Therapy Trial, Dies In US - NDTV

DetailsCategory: DNA RNA and CellsPublished on Friday, 16 October 2020 13:44Hits: 574

- Study targets a chronic autoimmune skin disorder affecting approximately 50,000 patients in the U.S. -

EXTON, PA, USA I October 15, 2020 I Castle Creek Biosciences,Inc, a privately-held, clinical-stage cell and gene therapy company leveraging its proprietary fibroblast technology platform to develop and commercialize innovative personalized therapies for underserved disorders with high unmet medical needs,announced that the first adult patient has been dosed in a Phase 1/2 clinical trial evaluating FCX-013, the company's investigational gene therapy, for the treatment of moderate to severe localized scleroderma.

"Dosing the first patient is an important milestone in the clinical development program for FCX-013, which we believe has the potential to be the first therapy to treat excessive collagen deposition at the site of localized scleroderma lesions in the skin and soft tissue," said John Maslowski, Chief Executive Officer of Castle Creek Biosciences. "Our hope is to relieve the debilitating, painful impact of localized scleroderma in patients who currently have limited treatment options."

Localized scleroderma is a chronic autoimmune skin disorder that leads to the excess production of collagen and causes thickening of the skin and connective tissue. In moderate to severe forms of the disorder, patients can experience discomfort, tightness and pain that limits their ability to function. Approximately 50,000 patients in the U.S. have moderate to severe localized scleroderma. Current treatment options include systemic or topical corticosteroids that target inflammation, UVA light therapy, and physical therapy. There are no U.S. Food and Drug Administration (FDA) approved therapies for patients living with this disorder.

"Localized scleroderma may be characterized based on the depth and pattern of lesions, and there are currently few treatment options to address the excessive collagen accumulation in the skin and connective tissue," said Mary Spellman, M.D., Chief Medical Officer of Castle Creek Biosciences. "With our proprietary fibroblast technology, we have an opportunity to develop and evaluate new personalized therapies that are designed for durability and formulated to be compatible with each patient's unique biology."

The open label, single cohort Phase 1/2 clinical trial is evaluating the safety of FCX-013 as its primary objective. Secondary objectives include assessments of fibrosis at targeted sclerotic lesions at various time points through 26 weeks post-administration of FCX-013.The trial will enroll up to 10 patients with moderate to severe localized scleroderma. More information about the Phase 1/2 trial is available at ClinicalTrials.gov and searching the identifier NCT03740724.

Castle Creek Biosciences is manufacturing FCX-013 at its in-house, current good manufacturing practices (cGMP), commercial-scale facility located in Exton, Pennsylvania.

About FCX-013

FCX-013 is Castle Creek Biosciences' investigational gene therapy candidate for the treatment of moderate to severe localized scleroderma. FCX-013 is an autologous fibroblast genetically modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 incorporates a biologic switch activated by an orally administered compound to control protein expression at the site of the localized scleroderma lesions. FCX013 is designed to be injected intradermally at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation. FCX-013 has been granted Orphan Drug, Rare Pediatric Disease and Fast Track designations by the FDA.

About Castle Creek Biosciences

Castle Creek Biosciences, Inc. is a privately-held, clinical-stage cell and gene therapy company advancing innovative personalized therapies for underserved disorders with high unmet medical needs. The company is using its proprietary fibroblast technology platform to develop D-Fi (debcoemagene autoficel, formerly designated FCX-007), an investigational gene therapy for the localized treatment of wounds in dystrophic epidermolysis bullosa (DEB). The company is also developing FCX-013, an investigational gene therapy for the treatment of moderate to severe localized scleroderma. The company operates an in-house, current good manufacturing practices (cGMP), commercial-scale facility located in Exton, Pennsylvania. Castle Creek Biosciences is a portfolio company of Paragon Biosciences. For more information, visit castlecreekbio.comor follow Castle Creek on Twitter @CastleCreekBio.

About Paragon Biosciences

Paragon is a life science innovator that creates, invests in and builds life science companies in artificial intelligence, cell and gene therapy, synthetic biology and biopharmaceuticals. The company's current portfolio includes Castle Creek Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the replicable Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

SOURCE: Castle Creek Biosciences

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Castle Creek Biosciences Announces First Patient Dosed in Phase 1/2 Clinical Trial of FCX-013 Gene Therapy for Treatment of Moderate to Severe...

The energetic 3-year-old boy toddled around the living room of his Longview home with a blanket over his head, announcing he was a ghost.

Finn Florczykowskis mother explained that he is excited for Halloween since he is now old enough to be aware of the holiday.

Over time, Finn will lose the ability to walk and also lose mobility in his arms and legs due to Duchenne muscular dystrophy, a rare and aggressive genetic disease that causes rapid muscular deterioration.

Hes energetic, sassy, and hes so smart, Finns mother, Paula, said, laughing. Hes everyones baby, so hes super spoiled.

Finn pulled blankets out of a cupboard, throwing them over his head to play ghost and attack his parents.

We want everyone to know his name, Paula said, picking him up.

Finn was born Sept. 13, 2017, and diagnosed with the disease when he was about 9 months old.

He had no symptoms, Paula said. He was in the NICU for high liver enzymes, which is not a sign. And he had some esophagus issues, which is also not a sign, but the pediatrician told us we should go and test him.

Paula was not worried about the test, she said.

I was in the Target parking lot when she called me, Paula said. It was devastating.

Everything changed that day on the drive back to their former home in Diana, she said.

Our perspective of how short and how terminal we all are, Paula said. Ive always said from day one, were all terminal well never know when were going to go.

Since Finns diagnosis, the familys life slowed down, especially during the pandemic.

Its been a blessing, I mean, weve grown closer as a family, Paula said, laughing. Weve just cherished every moment, and weve counted every hair on his head.

Finn has an older sister and brother, neither with the same condition.

This isnt like hes got a little cold or something, said his father, Dan Florczekowski.

DMD is incurable and irreversible. The disease typically affects boys and is similar to ALS in adults.

Finn is not showing any delays or developmental issues other than some calf swelling, Paula said.

You look out 10 years, its hard, its not easy to stomach, Dan said. They look great and then they end up losing mobility around 8 to 12 years old. Then between, usually around age 16 is when the deterioration really hits their lungs and hearts.

The disease eventually destroys the muscles that control the heart and lungs, which makes it fatal.

Thats the hard part to talk about, Dan said. Fifteen or 20 years ago, getting this was a death sentence. You were basically told by specialists to go home and love on your kid for 20 years, 18 to 24 years.

Dan added that through advancements in treatment and a comprehensive approach, the average lifespan for a child with DMD is extended by 10 years.

Hopefully, the next thing is gene therapy treatments, Dan said. They might go out even farther than that. We hope.

Finn sees a specialist in Massachusetts and is preparing for aggressive steroid treatment in an attempt to slow the disease.

They want us to do a gene therapy trial next September, Paula said. In order for him to qualify for that trial, he has to be on steroids for six months.

The plan is to go to Boston in February and begin the steroid treatments.

Were fighting time, Paula said.

Paula and Dan noticed that many people did not know about the disease or only knew a little about muscular dystrophy in general. The family wants to raise awareness of the condition and do what they can to raise funds for research into treatment and a cure.

From day one, when he was diagnosed, we very much knew we were going to start a nonprofit, Paula said.

They started Finns Friends as a nonprofit organization and started fundraising by selling bracelets and T-shirts.

The support from the community for his little organization has been overwhelming, Dan said. I got home last night and there was a $1,000 check in the mail. A donation.

The couple joined with and donates to Team Joseph, a nonprofit organization in Detroit that funds research to find a treatment or cure for DMD.

The first Finns Friends Golf Tournament is set Saturday at Tempest Golf Club in Gladewater. Part of the funds raised by the tournament will go to Team Joseph, which also provides a family assistance program.

We definitely, 100 percent, know that we have been super blessed with being able to go and see the best doctors and specialists, Paula said. Weve met families who cant even afford a mattress, you know, and so our heart has been helping basic needs children who have Duchenne and their families.

What will Finn be doing during the tournament?

Just telling everybody what to do, Paula said, smiling. Chasing everybody with his ghost costume.

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Longview family rallies around 3-year-old with rare, fatal genetic condition - Longview News-Journal

PARIS, France: A five-year-old girl with a rare neurodegenerative disease died in the US while taking part in a gene therapy trial run by French biotechnology company Lysogene, the firm said on Thursday.

The little girl was suffering from Sanfilippo syndrome or mucopolysaccharidosis type III, a rare genetic disease that alters brain development after birth and leads to premature death. In a statement Lysogene, a company developing gene therapy for central nervous system diseases in children, said "the immediate cause of death is currently unknown" and that there was as yet "no evidence that the event is linked to the study drug administration".

It said it was "profoundly saddened by the passing of this child" and was collecting "additional information" about the circumstances. The share price of the company dropped 19 percent to 2.05 euros in morning trading in Paris.

The girl was one of 19 people being treated in the trial conducted at eight hospitals in Europe and the United States. She died at home several months after receiving the therapy, consisting of a single injection, at one of four treatment sites in the US, Lysogene told AFP. In its statement the company said it was following the remaining 18 patients closely and remained "committed to the LYS-SAF302 development program".

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Child dies in US during gene therapy trial - The News International

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Key Market Insights

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Tag: Gene Therapy - The Think Curiouser

Immuno-oncology and CAR T cells energized the field of regenerative medicine, but for cell and gene to deliver on their promises, new, disruptive technologies and new modes of operation are needed. Specifically, that entails improving manufacturing to control variables and thus ensure product consistency, and maturing the regulatory environment to improve predictability.

Manufacturing cells is not like manufacturing small molecules, Brian Culley, CEO of Lineage Cell Therapeutics, told BioSpace. For cell therapy products to mature into real products that deliver on the promises of 10 years ago, they must be scalable which drives affordability and they must solve their purity issues.

On the clinical side, cell and gene therapies must find places where small molecules, antibodies or other traditional approaches may not be the best option.

For example, The era of transplant medicine is unfolding before us, Culley said. Because of the transplant component, cell therapy may enable changes the body never could do alone.

Lineage is addressing dry AMD and spinal cord injuries with two of its therapeutics.

Our approach is fundamentally different from traditional approaches. We replace the entire cell rather than modulate a pathway. There is a rational hypothesis where cell therapy can win, but first we need to fix the operational hurdles, Culley said.

To address the manufacturing challenges, Culley said, We work only with allogeneic approaches. For us, not being patient-specific is a huge advantage.

Not long ago, the industry was focused on 3D manufacturing in bioreactors.

Were beyond that, Culley said. For our dry AMD product, we can manufacture 5 billion retinal cells in a three liter bioreactor. The advantage is that the cells exist in a very homogenous space and are 99% pure.

As a result, they are more affordable and can be harvested with little manipulation.

Manual manipulation affects gene expression, he pointed out, so minimizing that, as well as the vast quantities of plastics typically required, results in a more controlled process and a more consistent product.

Additionally, Lineage introduced a thaw and inject formulation, so the cell therapy can be thawed in a water bath, loaded into a chamber and injected, all within a few minutes. Traditional dose administration requires washing, plating and reconstituting the cells the before they are administered to a patient.

Getting rid of the prior day dose prep is one example of the maturation of the field, which we are deploying today to help usher in a new branch of medicine, Culley said.

At Lineage, were tackling problems that largely were intractable. For dry AMD, theres nothing approved by the FDA. No one know why the retinal cells die off, so we manufacture brand new retinal cells (OpRegen) and implant them, Culley said. Were seeing very encouraging clinical signs, including the first-ever case of retinal restoration.

Retinal cells compose a thin layer in the back of the eye, Culley explained.

They start to die off in one spot, and that area grows outward. When we inject our manufactured cells where the old ones died, weve seen the damaged area shrink and the architecture in previously damage areas completely restored, Culley said. Weve treated 20 patients for dry AMD in, ostensibly, safety trials, but you cant help but notice efficacy when a patient reads five more lines on an eye chart. Its hard to imagine our intervention wasnt responsible for that, especially when humans cant regenerate retinal tissue.

The spinal injury program (OPC1) may represent an even greater breakthrough. As with dry AMD, there is no FDA-approved therapy.

We manufacture oligodendrocytes and transport them into the spinal cord, to help produce the myelin coating for axons, he told BioSpace. Because of the oligodendrocytes, the axons grow, become myelinated, and begin to function. Small molecule and antibody therapies havent been able to do that.

So far, 25 people have been treated in a Phase I/II trial. Culley reported cases in which a quadriplegic man, after OPC1 therapy, is now typing 30 to 40 words per minute, and another who now can throw a baseball. Its not unusual for patients who initially were completely paralyzed to now schedule their treatments around college classes, Culley said.

Humans can have varying degrees of recovery from spinal cord injury, but these are higher than we would expect, Culley said.

Other cell and gene companies are advancing solutions, too.

Many companies with induced pluripotent stem cells (iPSCs) are trying to figure out how to get scalability, purity, and reproducibility to work for them. Its not a quick fix, he said.

One of the challenges is balancing the clinical and manufacturing aspects of development.

If you have a technology thats not yet commercially viable, but you have clinical evidence, its tempting to focus on the clinical side, Culley said.

Too many companies do that, and then find their candidate must be reworked for scale up. Therefore, consider scale up and manufacturing early.

Theres a need for balance at a more granular level, too. For example, he asked, How many release criteria do you need? Just because you know a cell expresses a certain surface marker, does that add to your process? Ive seen companies ruined by trying to be perfect, and others by rushing headlong, seeing evidence where evidence doesnt exist.

As Lineage matures its processes to support larger clinical trials, the greatest challenges have been time It takes 30 to 40 days to grow cells, Culley said and regulatory uncertainty. Often, there is no regulatory precedence so there are holes to be addressed. For example, cell and gene therapies sometimes have a delivery component such as a scaffold or delivery encapsulation technology that also must be considered. Real-time regulatory feedback isnt available, so you proceed, presuming that what youre doing will be acceptable to regulators.

The FDA recognizes that new, disruptive technologies and approaches are being used, and must be used, for cell and gene therapy to reach patients.

The FDA is responsive and is trying to push guidance out, Culley said, but it takes time.

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Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation - BioSpace

Global Gene Therapy for Inherited Genetic Disorders Market: Overview

Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer.

Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.

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Global Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease).

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practicaland accessiblegene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations.

Some of the bigplayerseyeing promising stakes in the gene therapy for inherited genetic disorders market areSpark Therapeutics Inc., Orchard Therapeutics, Novartis AG, bluebird bio Inc., and BioMarin Pharmaceutical.

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Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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Gene Therapy for Inherited Genetic Disorders Market Robust pace of Industry during 2018-2028 - The Think Curiouser

Ori Biotech will use $30 million from a Series A financing round to finalize and test its closed and automated cell and gene therapy technology.

In January, Ori Biotech came out of stealth mode through a $9.4 million seed round, introducing the cell and gene therapy space to its technology platform, which aims to significantly reduce the cost of production of such therapies.

Nine months on and the firm has raised a further $30 million in a Series A financing round led by Northpond Ventures, a global science, medical, and technology-driven venture fund, with European venture fund Octopus Ventures.

Image: iStock/kgtoh

The money will be used to finalize the development of the platform, test the MVP [minimum viable product] with our early access customers, hire additional staff and bring the platform to market in 2022, a spokesperson from the firm told Bioprocess Insider.

The platform is based on a flexible and scalable bioreactor and fluid handling system, which could revolutionize the advanced therapy space, including reducing the cost of goods sold (COGS) for autologous therapies by as much as 60-80%, the firms CEO Jason Foster previously told this publication.

The firm teamed up with contract development and manufacturing organization (CDMO) Minaris Regenerative Medicine, formerly known as Hitachi Chemical Advanced Therapeutics Solutions (HCATS), earlier this year.

The partnership is progressing well and they have been testing the first generation system in their processes and generating data comparing the Ori platform to existing first generation technologies, we were told.

Furthermore, Ori has teaming up with numerous end users to bring their technology platform to fruition.

We have announced partnerships with Achilles Therapeutics and several other unannounced partnerships with therapy developers are kicking off as we speak.

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Ori looks to advance closed cell therapy tech - BioProcess Insider

SAN DIEGO, Oct. 19, 2020 (GLOBE NEWSWIRE) -- Histogen, Inc.(NASDAQ: HSTO), a clinical-stage therapeutics company focused on developing potential first-in-class restorative therapeutics that ignite the bodys natural process to repair and maintain healthy biological function, today announced the appointment of Moya Daniels as Executive Vice President and Head of Regulatory, Quality and Clinical Operations.

I am pleased to have Moya join the Histogen team at this important time as we continue to progress our pipeline candidates in the clinic, said Richard W. Pascoe, President and Chief Executive Officer of Histogen. Moya has over 30 years of experience in the biotechnology and pharmaceutical industries and most notably has extensive expertise in the cell and gene therapy field. Moreover, Moya brings an impressive track-record to Histogen as an industry leader which will complement our existing senior management team.

Ms. Daniels most recently served as Senior Vice President of GMP Quality at SanBio, where she successfully led the CMC regulatory development and GMP Quality function in support of the planned Japan commercialization of their lead product candidate. Prior to SanBio, Ms. Daniels was the Senior Vice President of Regulatory Affairs and Global Quality Assurance at Orchard Therapeutics, where she led the companys CMC regulatory and GXP quality strategy and was part of the team that led the in-licensing of GSKs rare disease gene therapy portfolio. Prior to joining Orchard, Ms. Daniels served as Vice President of Regulatory Affairs and Quality Assurance at Fate Therapeutics, Inc, where she led the development of the global regulatory strategy and quality assurance function and was interim head of Clinical Operations. Moya led development of the global regulatory strategy, quality, and global clinical operations at Osiris Therapeutics, which led to the approval of Prochymal, the first approved allogeneic cell therapy indicated for the treatment of pediatric steroid refractory acute Graft Versus Host Disease in Canada and New Zealand. Ms. Daniels also held a senior leadership position at Macrocure as Vice President of Global Regulatory Affairs and currently serves as a Scientific Advisory Board member for Indapta Therapeutics.

I am energized by Histogens leadership team and scientists and look forward to working with them to advance the companys technology through clinical development. Histogens proprietary manufacturing process that generate biological materials that stimulate a patients own stem cells to activate and regenerate tissue is truly unique, said Ms. Daniels. Histogens focus on developing potential first-in-class restorative therapeutics is perfectly aligned with my industry experience and scientific passion.

About HistogenHistogen Inc. is a clinical-stage therapeutics company focused on developing potential first-in-class restorative therapeutics that ignite the bodys natural process to repair and maintain healthy biological function. Histogens innovative technology platform utilizes cell conditioned media and extracellular matrix materials produced by hypoxia-induced multipotent cells. Histogens proprietary, reproducible manufacturing process provides targeted solutions across a broad range of therapeutic indications including hair growth, dermal rejuvenation, joint cartilage regeneration and spinal disk repair. For more information, please visit http://www.histogen.com.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. For example, we are using forward-looking statements when we discuss our future operations and our ability to successfully initiate and complete clinical trials, obtain clinical trial data and achieve regulatory milestones and related timing; the nature, strategy and focus of our business; the sufficiency of our cash resources and ability to achieve value for our stockholders; and the development and commercial potential and potential benefits of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on our current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of ours that could differ materially from those described in or implied by the statements in this press release, including: the uncertainties associated with the clinical development and regulatory approval of our product candidates, including potential delays in the commencement, enrollment and completion of clinical trials; the potential that earlier clinical trials and studies of Histogens product candidates may not be predictive of future results; risks related to business interruptions, including the outbreak of COVID-19 coronavirus, which could seriously harm our financial condition and increase its costs and expenses; the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all; and the ability to attract and retain key personnel. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including those risks discussed in our filings with the Securities and Exchange Commission. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events, or circumstances or otherwise.

CONTACT:

Susan A. KnudsonExecutive Vice President & CFOHistogen Inc.ir@histogen.com

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Histogen Appoints Moya Daniels as Executive Vice President and Head of Regulatory, Quality and Clinical Operations - BioSpace

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donors immune cells attack the recipients cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patients own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death, said Gary Fortin, head of severe genetic disease programmes at Bluebird.

If approved, eli-cel would represent the first therapy for CALD that uses a patients own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor, he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebirds development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum

PLEASANTON, Calif., Oct. 6, 2020 /PRNewswire/ -- Unchained Labs, the life sciences company that's all about getting researchers the right tool for the job, launched its new gene therapy quantification application on Stunner today. Stunner is the first platform that gives researchers a full read-out on how many AAV capsids they have, how many are packed with DNA and if there is aggregation in less than a minute using only 2 L of sample.

Adeno-associated viruses (AAVs) used in gene therapies are hard to manufacture and tough to characterize. Researchers need to be able to quickly and accurately measure their capsid titer, and how many of them are full of DNA they are supposed to deliver to cells. Other methods that gather this info require labels, dyes, reagents, standard curves or use a lot of sample. Pulling it all together is a big lift and takes way too long with existing technologies.

Stunner makes AAV titer a snap. Researchers just pipette 2 L of their sample, up to 96 samples per run, and then Stunner takes it from there. UV/Vis measures the total protein and DNA in the sample. Dynamic light scattering identifies AAV capsids based on their size and checks for aggregation. Stunner then combines all this information to provide an accurate titer and empty/full ratio.

"Gene therapy researchers are hungry for better tools and Stunner delivers all the data they need," said Taegen Clary, VP of Marketing at Unchained Labs. "No other instrument quantifies AAV capsids, both full and empty, and can see aggregates. These complex molecules demand that traditional techniques be reimagined or combined to provide better insights, and with Stunner's AAV application we've done both."

About Unchained LabsHere's the deal. We're all about helping researchers break free from tools that just don't cut it. Unleashing problem-tackling products that make a huge difference in the real science they do every day. That's our mantra, our promise and we own it. We're located in Pleasanton, CA and can be found online at http://www.unchainedlabs.com.

Contact:Taegen ClaryVP of Marketing, Unchained Labs[emailprotected]925.587.9806

SOURCE Unchained Labs

http://www.unchainedlabs.com

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Unchained Labs launches the ultimate gene therapy tool; tackle AAV titer, empty/full ratio and more with Stunner! - PRNewswire

CSO C. David Pauza, Ph.D. Presents on Cell and Gene Therapy for HIV Disease

Chief Science Officer C. David Pauza, Ph.D. Presents At the Intersection of Genetic Medicine and Immunotherapy: Clinical Experience with a Cell and Gene Therapy for HIV Disease

ROCKVILLE, Md., Oct. 08, 2020 (GLOBE NEWSWIRE) -- AmericanGene Technologies(AGT)a cutting-edge cell and gene therapy company in Rockville, Maryland announced today that CEO Jeff Galvin will present at the annual Cell & Gene Meeting on the Mesa. The meeting will be held virtually October 12-16. Galvins presentation will highlight the company's technology, including AGT103-T, a therapeutic intended to cure HIV, which is scheduled to begin Phase 1 clinical trials this month.

Specifics of AGTs clinical trial can be found at https://clinicaltrials.gov/ct2/show/NCT04561258, and details of the HIV therapy intended to cure the disease are on the AGT web site.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a five-day virtual conference featuring more than 120 presentations from the leading public and private companies. These presentations will highlight the most exciting technical and clinical achievements from the past 12 months in cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies. The meeting also includes over 100 panelists and features speakers taking part in 20 in-depth sessions covering all aspects of cell and gene therapy commercialization.

The following are specific details regarding AmericanGene Technologies presentation at the conference:

Please visit http://www.meetingonthemesa.com for full information including registration. Complimentary attendance at this event is available for accredited investors and members of the media. Investors should contact Laura Stringham at lstringham@alliancerm.org and interested media should contact Kaitlyn Dupont at kdupont@alliancerm.org. The event hashtag is #CGMOM20.

About American Gene Technologies (AGT)

AmericanGene Technologies(AGT)is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies. AGTs mission is to transform peoples lives by designing highly effective therapeutics to cure infectious diseases, cancers, and inherited disorders. AGT has received three patents for the technology used to make the AGT103-T cell product and ten patents for its uniqueimmuno-oncology approachto stimulategamma-delta () T cellsto destroy a variety of solid tumors. The company has also developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. AGT's treatment for PKU has been grantedOrphan Drug Designationby the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2021.

More information is available on:

Website - http://www.americangene.comLinkedIn - LinkedInTwitter -@americangeneFacebook -@amerigeneInstagram -@americangenetechnologies

American Gene Technologies Contacts:

C. Neil Lyons, Chief Financial OfficerPhone: (301) 337-2269Email:info@americangene.comwww.americangene.com

Sasha Whitaker, Digital Marketing and CommunicationsPhone: (301) 337-2100Email:swhitaker@americangene.comwww.americangene.com

A video is available at the following link:https://www.globenewswire.com/NewsRoom/AttachmentNg/2623868c-a33f-49c1-bc46-0c114cbd1d83

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American Gene Technologies to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

As with many conferences, the Cleveland Clinics 2020 Medical Innovation Summit went virtual this year. Still, the event featured the hotly anticipated top 10 list of innovations for 2021 that saw a range of therapies, including options for arrhythmias and gene therapy for hemoglobinopathies.

Usually, the event sees all innovations presented in a single session, but the top three were highlighted on their own.

Ranked in order of expected importance, the list was led by gene therapy for hemoglobinopathies, which are genetic disorders affecting the structure or production of the hemoglobin molecule. The most common hemoglobinopathies include sickle cell disease and thalassemia. Now, there is an experimental gene therapy that could help patients make functional hemoglobin molecules.

This is a genetic disease that affects more than 20 million people worldwide, said Rabi Hanna, department chair, pediatric hematology, oncology, & blood & marrow transplantation in highlighting the problem. Of note, sickle cell disproportionally affects the Black community. Only three medications have been approved in the past 50 years.

If adopted, this therapy could lead to a cure a very dramatic development. Hanna noted that many companies are in phase III, with one applying for approval and receiving breakthrough designation. He hopes the U.S. FDA could recognize such candidates in the next year.

That was followed by a medication for primary-progressive multiple sclerosis (MS), which affects about 15% of people with the disease. The FDA has approved a therapeutic monoclonal antibody with a novel target, namely the primary-progressive population.

For the first time we have treatment for all forms of MS, explained Adrienne Boissy, chief experience officer at the Cleveland Clinic.

Patients with this form of MS have not had a treatment in the past. This specific treatment is meant to impact the disease itself, both the MRI appearance in this disease as well as functional measures that matter for the patient, like walking and being able to maintain the life that they want to have at the fullest with this treatment.

Coming in third was smartphone-connected pacemaker devices, which are used to prevent or correct arrhythmias. These innovations aim to help in the disconnect between patients and their cardiac treatment. When paired with a mobile app, patients can gain greater insight into health data from the pacemakers. At the same time, the devices can transmit health information to physicians.

Around the world, we have millions of patients walking with pacemakers and defibrillators, and its important to know that remote monitoring of these devices is an essential part of the care of these patients, explained Khaldoun Tarakji. Much data shows that having this leads to better outcomes, perhaps through the early detection of arrhythmias. Still, adoption and adherence have remained suboptimal, he noted.

Typically, remote monitoring has taken place via bedside monitor. Patients must be in close contact with these devices. Now, a smartphone or tablet can act as that monitor, transmitting data from anywhere.

Following that innovation was a treatment for cystic fibrosis, which is caused by a defective cystic fibrosis transmembrane conductance regulator (CFTR) protein. A class of drugs called CFTR modulators correct the proteins action, but medications developed before 2019 only were effective in a subset of people with certain mutations. However, last October, the FDA approved a new option that provides relief for patients with the most common CF gene mutation (F508 del), which is estimated to represent 90% of individuals living with the disease.

Elexacaftor/tezacaftor/ivacaftor is a fixed-dose combination medication of CFTR modulators and chloride channel openers approved for use in those patients with CF who have at least one F508del mutation in the CFTR gene.

Definitely a game changer, confirmed Elliott Dasenbrook, director, Adult Cystic Fibrosis Program, Cleveland Clinic, when asked about the class of drugs. This new class of medications the modulators help the protein channel actually, its misfolded as a result of the genetic mutations, so it helps correct that misfolding and it also helps move it throughout the cell so that it can get to the membrane, where it actually functions.

Holding the number five spot was a universal hepatitis C treatment. While this disease has serious consequences, there is no vaccine, and patients must take medication. Many treatments led to adverse side effects or only were effective for certain genotypes of the disease. However, a fixed-dose combination medication holds hope for these patients. More than 90% effective for hepatitis C genotypes one through six, the therapy represents an effective option for a wider scope of patients.

Coming in next is bubble CPAP (b-CPAP) for increased lung function in premature babies. Premature babies with infant respiratory distress syndrome (IRDS) often require ventilation. Those with IRDS commonly are administered a surfactant during mechanical ventilation, a practice that can cause lasting lung injury. B-CPAP is a noninvasive ventilation strategy, delivering continuous positive airway pressure to newborns to maintain lung volumes during exhalation.

The procedure can stimulate the lung to grow while minimizing the injury to the lung, explained Hany Aly, department chair, neonatology, Cleveland Clinic.

Unsurprisingly, telemedicine, which played a big role during the ongoing COVID-19 pandemic, made the list. Specifically, the clinic highlighted state and national regulators actions in reducing telehealth adoption barriers. Their actions permitted more widespread telehealth adoption.

COVID was a complete disruptor, and it was a disruptor that we needed, said Beri Ridgeway, associate chief of staff & chair, obstetrics, Gynecology & Women's Health Institute, Cleveland Clinic. Policy is so critical, and I believe it was one of the barriers that prevented us from taking telehealth to the next level. By taking steps on the state and federal levels in March to remove these barriers, patients benefitted. Changes came in terms of who could see patients, by what platform to include chat functions on the phone and coverage. By April, 90% of clinicians were using some sort of telehealth, it was noted.

The number eight spot was held by a vacuum-induced uterine tamponade device for postpartum hemorrhage, which affects about 1% to 5% of women who give birth. Those who experience this condition face possible blood transfusions and emergency hysterectomy. Nonsurgical interventions have involved balloon devices that expand the uterus while compressing the site of bleeding. However, a vacuum-induced uterine tamponade has caught the attention of the medical community. It is a low-tech solution that is potentially translatable to developing countries with low resource availability.

I think this is going to replace the use of the balloon compression devices for control of hemorrhage, said Edward Chien, department chair, obstetrics & gynecology, Cleveland Clinic.

In September, Alydia Health said it had received U.S. FDA clearance of the Jada system, which is designed to rapidly control and treat postpartum hemorrhage and abnormal postpartum uterine bleeding. At the same time, it announced the publication of its pivotal PEARLE study in Obstetrics & Gynecology, which demonstrated that Jada is safe and highly effective in using vacuum to control postpartum bleeding.

Holding down the ninth spot is PARP inhibitors for prostate cancer, a condition one in nine men will be diagnosed with in his lifetime. PARP inhibitors pharmacological inhibitors for cancer treatment block proteins called PARP that help repair damaged tumor DNA in individuals with BRCA1 and BRCA2 gene mutations.

The PARP inhibitors rucaparib and olaparib have been demonstrated to delay the progression of prostate cancer in men with refractory cancer and BRCA mutations. Both were approved for the prostate cancer indication in May.

While not a new treatment in cancer overall, PARP inhibitors could prove very helpful for men with this condition. And while the gynecological field has led the way, this option has been considered for years in prostate cancer, said Moshe Ornstein, associate staff, Hematology & Medical Oncology Cleveland Clinic. They will be appropriate for patients who have specific mutations in their DNA damage repair pathways.

Finally, coming in at number 10 was immunologics for migraine prophylaxis. While a host of products have tried to help this condition, nothing was directed solely for migraines. In 2018, new medications were developed for migraine sufferers that blocked the activity of a molecule called calcitonin gene-related peptide. This medication is the first to be specifically designed for the preventive treatment of migraine.

The first three medications approved for the prevention of both chronic and episodic migraines are erenumab, galcanezumab, and fremanezumab. These options were approved by the FDA in May and September 2018, respectively. They are given as once-monthly injections and are extremely well-tolerated. For most people, there are few adverse reactions aside from some pain at the injection site.

Its an exciting time to be in headache medicine. Its been years since weve had any type of new therapeutics to treat this disorder, MaryAnn Mays, director, Neurology Residency Program at the Cleveland Clinic, said.

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Smartphone-connected pacemaker devices, experimental gene therapy among top 10 innovations at annual event - BioWorld Online

FREIBURG, Germany, October 08, 2020 / B3C newswire / -- CellGenix GmbH, a pioneer for GMP grade reagents and a leading global supplier of high quality raw and ancillary materials for cell and gene therapies, announces the completion of the second phase of their facility expansion. CellGenix has reconstructed and expanded their production facility for cytokines and implemented a state-of-the art automated filling and freeze-drying line.

By implementing an automated filling and freeze-drying line and increasing bulkware production footprint, finished product capacity for cytokines is substantially increased. This continues to enable CellGenix to meet the fast-growing demand for critical raw and ancillary materials as more customers reach late-stage clinical development and commercialization of their cell and gene therapies. Scaling up from phase I or II clinical trials to large-scale commercial manufacturing, the demand for GMP cytokines often rises by several magnitudes. Consequently, a secure supply chain is imperative. A delay in delivery or change in product quality could lead to delays in production, not only increasing costs but also putting precious patient samples in jeopardy.

The newly designed and expanded facility enables CellGenix to streamline their production processes. The automated filling process also provides increased accuracy and reduced risks, which further increases the safety of their preclinical and GMP cytokines. The automated filling, stoppering and capping system (operates under an Open Restricted Area Barrier System (ORABS) and) is compliant with EU GMP Annex 1 for the manufacture of sterile medicinal products, also taking into account the new version which is currently under revision.

In a first expansion phase in 2018 CellGenix added additional space and personnel in its quality control, R&D, logistics and warehouse departments. By finishing both expansion phases, CellGenix has further strengthened their position as a leading supplier for large scale manufacturing in the cell and gene therapy space.

The expansion of our facility is a result of the robust, sustainable, and profitable growth, generated by long standing and trustful customer relationships we achieved over many years. CellGenix is now well prepared for the future growth of the cell and gene therapy market. It supports our goal to further establish CellGenix as a key provider in the fight against disease, preferred supplier or raw and ancillary materials and trusted partner for large scale manufacturing of cell and gene therapies. Felicia Rosenthal, Chief Executive Officer at CellGenix.

About CellGenixCellGenix is a leading global supplier of high quality raw and ancillary materials for the expanding market of cell and gene therapy and regenerative medicine. CellGenix develops, manufactures and markets human cytokines, growth factors, and other recombinant cell culture components in preclinical and GMP quality as well as proprietary serum-free media for further manufacturing of ATMPs. As a former ATMP developer and manufacturer, in more than two decades CellGenix gained in-depth cell processing knowledge and superior regulatory expertise. With this unique background, CellGenix understands the high requirements their customers are facing during product development and the regulatory approval process. To meet the increasing demand of GMP quality raw materials for ATMP manufacturing CellGenix has recently expanded manufacturing capacity, built additional R&D and QC laboratories, and warehouse space. The upgrade also introduced state-of-the-art, automated, large-scale capacities for recombinant protein products in the existing GMP facilities. CellGenix is headquartered in Freiburg, Germany and operates a subsidiary near Boston in Portsmouth, USA.

Contact

CellGenixFelicia M. RosenthalCEOThis email address is being protected from spambots. You need JavaScript enabled to view it. Am Flughafen 1679108 Freiburg, Germany

Keywords: Cytokines; Genetic Therapy; Cell- and Tissue-Based Therapy; Regenerative Medicine; Culture Media, Serum-Free; Indicators and Reagents; Manufactured Materials; Cell Culture Techniques; Freeze Drying

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CellGenix Completes Facility Expansion to Stay Ahead of the Increasing Demand for GMP Raw Materials - b3c newswire

The innovation experts at Cleveland Clinic have peered into their crystal ball and pronounced the most profound medical advancements that will impact the care and treatment of patients in the coming year.

During the closing session of the 2020 Cleveland Clinic Medical Innovation Summit, the 18th year the conference has been held, Cleveland Clinic Innovations shared its annual list of top 10 medical innovations. The breakthrough technologies were selected by a committee of Cleveland Clinic subject matter experts, led by Will Morris, MD, executive medical director, Cleveland Clinic Innovations, and Akhil Saklecha, MD, managing director, Cleveland Clinic Ventures.

1. Gene Therapy for Hemoglobinopathies

Experimental gene therapy gives new hope to those suffering from hemoglobinopathies, genetic disorders responsible for sickle cell disease and thalassemia. Through treatment, those with these conditions have the potential ability to make functional hemoglobin molecules, reduce the presence of sickled blood cells or ineffective red blood cells in thalassemia, and prevent associated complications.

2. Novel Drug for Primary-Progressive Multiple SclerosisA new, U.S. Food and Drug Administration (FDA)-approved therapeutic monoclonal antibody with a novel target is the first and only multiple sclerosis (MS) treatment for 15% of primary-progressive patients, who experience gradual onset and steady progression of signs and symptoms. In those with MS, the immune system attacks the fatty protective myelin sheath that covers the nerve fibers, causing communication problems between the brain and the rest of the body that can result in permanent damage or deterioration and eventual death.

3. Smartphone-Connected Pacemaker Devices

Remote monitoring of implantable pacemakers and defibrillators traditionally occurs through a bedside console that transmits data to the physician. Yet many patients don't understand how it functions, and adherence to remote monitoring has been suboptimal. New Bluetooth-enabled pacemaker devices used in conjunction with a mobile app, offer a convenient new method to transmit data, while also providing patients greater insight into their health status.

4. New Medication for Cystic Fibrosis

A new combination drug, approved by the FDA in October 2019, provides relief for patients with the most common cystic fibrosis gene mutation (F508 del), estimated to represent 90% of individuals living with the disease. Medications developed prior to last year had only been effective in a subset of people with certain mutations.

5. Universal Hepatitis C Treatment

A new, approved fixed-dose combination medication has vastly improved hepatitis C treatment. More than 90% effective for hepatitis C genotypes one through six, the therapy represents an effective option for a wider scope of patients. With no vaccine for the hepatitis C virus, patients have been limited to medication, but many treatments were accompanied by adverse side effects or only effective for certain genotypes of the disease.

6. Bubble CPAP for Increased Lung Function in Premature Babies

Newborns with infant respiratory distress syndrome now have access to a safer method of ventilation though b-CPAP, a non-invasive ventilation strategy. Unlike mechanical ventilation, which administers a surfactant that can cause lasting lung injury, b-CPAP maintains lung volumes during exhalation through oscillating, rather than constant pressure, minimizes physical trauma, and stimulates lung growth when administered over a prolonged period.

7. Increased Access to Telemedicine through Novel Practice and Policy Changes

While telehealth technologies and initiatives have been around for years, COVID-19 forced them into the spotlight and stimulated widespread adoption. "Since March, state and federal regulators have moved quickly to reduce telehealth adoption barriers, understanding that these tools can speed access to care while protecting healthcare workers and community members," according to Cleveland Clinic. "These measures opened the floodgates for telehealth, allowing for new programs and the expansion of existing networks."

8. Vacuum-Induced Uterine Tamponade Device for Postpartum Hemorrhage

A low-tech solution is now part of the arsenal to fight postpartum hemorrhage (PPH). Vacuum-induced uterine tamponade uses negative pressure created inside the uterus to collapse the bleeding cavity, causing the muscle to close off the hemorrhaging vessels. The device represents another minimally invasive tool that is potentially translatable to developing countries with minimal resources.

9. Prostate Cancer PARP Inhibitors

Previously known for their success in treating womens cancers, two PARP inhibitors, which block proteins that help repair damaged tumor DNA in people with BRCA1 and BRCA2 gene mutations, were approved for prostate cancer treatment in May. These pharmacological agents have been demonstrated to delay the progression of prostate cancer in men with refractory cancer and DNA repair pathway mutations.

10. Immunologics for Migraine Prophylaxis

New medications were developed in 2018 help head off migraine pain by blocking activity of the calcitonin gene-related peptide (CGRP) molecule, which spikes during these headaches. In 2020 this FDA-approved class of medication became the first actively prescribed therapeutic for preventative treatment of migraines, which affect an estimated 12% of the U.S. adult population.

More information about the these innovations, including videos and year-by-year comparisons is available on the website.

Mandy Roth is the innovations editor at HealthLeaders.

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Cleveland Clinic Names Top 10 Medical Innovations; Sickle Cell Therapy Tops the List - HealthLeaders Media

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy BMN 307 (BioMarin Pharmaceutical) for the treatment of patients with phenylketonuria (PKU).

BMN 307 is an adeno-associated virus type 5 (AAV5)-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells.

The Company recently initiated an open-label phase 1/2 study (PHEARLESS) to evaluate the safety, efficacy and tolerability of a single intravenous administration of BMN 307 in adults with PKU with PAH deficiency. The study will assess whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.

Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy, said Hank Fuchs, MD, President, Worldwide Research and Development at BioMarin. We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit biomarin.com.

BioMarin, pioneer in phenylketonuria (PKU) and gene therapy, receives FDA Fast Track designation for PKU investigational gene therapy, BMN 307. https://www.prnewswire.com/news-releases/biomarin-pioneer-in-phenylketonuria-pku-and-gene-therapy-receives-fda-fast-track-designation-for-pku-investigational-gene-therapy-bmn-307-301144678.html. Accessed October 5, 2020.

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Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked - Monthly Prescribing Reference

BURLINGTON, Mass., Oct. 08, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc.(Nasdaq:FLXN) announced today thatAdam Muzikant, Ph.D., Senior Vice President, Business Development, will present at the annual 2020 Cell & Gene Virtual Meeting on the Mesa. Dr. Muzikant will provide a review of FX201, an investigational, intra-articular, IL-1Ra gene therapy product candidate in clinical development for the treatment of osteoarthritis (OA).

The Company is conducting an open-label, Phase 1 dose-escalation trial evaluating the safety and tolerability of FX201 in patients with knee OA. The trial is intended to test low, mid and high doses of FX201 in cohorts of five to eight patients. Following the completion of the low-dose cohort and data review by an independent Drug Monitoring Committee, the trial has advanced to enrolling the mid-dose cohort. Data from the study are anticipated in 2021.

Organized by theAlliance for Regenerative Medicine, the 2020 Cell & Gene Meeting on the Mesa will be delivered in a virtual format over the course of five days beginning on October 12. The conference will feature more than 120 presentations by leading public and private companies highlighting the technical and clinical achievements in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

About FX201FX201 (humantakinogene hadenovec) is a novel, clinical-stage, intra-articular gene therapy product candidate which utilizes a helper-dependent adenovirus (HDAd) vector based on human serotype 5 (Ad5) that is designed to transfer a gene to cells in the joint to produce an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), under the control of an inflammation-sensitive promoter. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of OA. By persistently suppressing inflammation, Flexion believes FX201 holds the potential to provide long-term pain relief and functional improvement, and to modify disease progression.

About Flexion TherapeuticsFlexion Therapeutics(Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with OA, the most common form of arthritis. The company's core values are focus, ingenuity, tenacity, transparency and fun. Visitflexiontherapeutics.com.

Forward-Looking Statements This release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; timing and plans with respect to the Phase 1 clinical trial of FX201; and the potential therapeutic and other benefits of FX201, are forward looking statements. These forward-looking statements are based on managements expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, the fact that the impacts and expected duration of the COVID-19 pandemic are uncertain and rapidly changing; the risk that we may not be able to maintain and enforce our intellectual property, including intellectual property related to FX201; risks related to clinical trials, including potential delays, safety issues or negative results; and other risks and uncertainties described in our filings with the Securities and Exchange Commission (SEC), including under the heading Risk Factors in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 filed with the SEC on August 5, 2020 and subsequent filings with the SEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contacts:

Scott YoungVice President, Corporate Communications & Investor RelationsFlexion Therapeutics, Inc.T: 781-305-7194syoung@flexiontherapeutics.com

Julie DownsAssociate Director, Corporate Communications & Investor Relations Flexion Therapeutics, Inc.T: 781-305-7137jdowns@flexiontherapeutics.com

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Flexion Therapeutics to Present at the 2020 Cell & Gene Virtual Meeting on the Mesa - GlobeNewswire

Detect Lysosomal Storage Diseases program reduces barriers to genetic diagnosis through sponsored testing for lysosomal storage disorders, including GM2 gangliosidosis

Behind the Seizure program can help accelerate genetic epilepsy diagnosis in children experiencing unprovoked seizures

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced a partnership with Invitae, a leading medical genetics company, to support Invitaes Detect Lysosomal Storage Diseases (Detect LSDs) and Behind the Seizure programs. The Detect LSDs program enables the rapid diagnosis of lysosomal storage disorders (LSDs), including GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff disease). The Behind the Seizure program is an innovative, cross-company collaboration that supports faster diagnosis for children with epilepsy. The Behind the Seizure program will also support patient identification across Tayshas broad pipeline of gene therapies for which a number of indications have an underlying seizure phenotype.

"Through both initiatives, we are supporting the rapid identification of patients with debilitating diseases, allowing them to gain access to earlier therapeutic interventions. For LSDs, there are more than 50 different disorders with overlapping symptoms, making misdiagnosis common," said RA Session II, Tayshas President, CEO and Founder. "Likewise, more than 50% of epilepsies have a genetic basis. When a patient presents with seizures, genetic testing may help identify more than 100 underlying, often rare conditions. We are proud to support these initiatives to help patients gain timely access to natural history studies, clinical trials, and ultimately disease-modifying therapies."

Eligible individuals suspected of having an LSD or epilepsy will gain access to genetic testing and counseling at no charge through these programs. The Detect LSDs program will help identify individuals who are eligible for Tayshas study evaluating TSHA-101 in patients with GM2 gangliosidosis, expected to enter the clinic later this year. The Behind the Seizure program will enable patient identification across Tayshas broad pipeline of indications, some of which have an underlying seizure phenotype, and rapid enrollment into natural history studies and clinical trials.

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"Increasing access to genetic testing can support earlier diagnosis of neurodegenerative diseases, which in turn enables clinicians to provide precision therapies sooner and better overall outcomes," said Robert Nussbaum, M.D., Chief Medical Officer of Invitae. "These unique, cross-company collaborations have been shown to help increase access to testing and reduce time to diagnosis. We are pleased Taysha has joined us in helping increase access to testing for children impacted by neurodegenerative conditions."

Additional details, as well as terms and conditions of the Detect LSDs program, can be found at https://www.invitae.com/en/detectLSDs/. To learn more about the Behind the Seizure program, please visit https://www.invitae.com/en/behindtheseizure/.

About Taysha Gene Therapies

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

About Invitae

Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the conduct or timing of our partnership with Invitae and our planned clinical trial of TSHA-101 for the treatment of GM2 gangliosidosis, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our prospectus dated September 23, 2020, as filed with the Securities and Exchange Commission ("SEC") on September 24, 2020, pursuant to Rule 424(b) under the Securities Act of 1933, as amended, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201006005450/en/

Contacts

Company Contact: Niren Shah, PharmD, MBATaysha Gene TherapiesNshah@tayshagtx.com

Media Contact: Carolyn HawleyCanale Communicationscarolyn.hawley@canalecomm.com

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Taysha Gene Therapies Partners with Invitae to Enable Rapid Access to Genetic Testing and Earlier Diagnosis of Patients with CNS Disease for Rare and...

Cancer Gene Therapy Market report offers in-depth analysis of the industry size, share, major segments, and different geographic regions, forecast for the next five years, key market players, and premium industry trends. It also focuses on the key drivers, restraints, opportunities and industry challenges.

Growing prevalence of cancer with rising mortality rates will augment cancer gene therapy industry forecast in the coming years. Cancer therapies incorporated with genetically modified genes ai in blocking the growth of the tumors.

Efficient PCR and isothermal amplification technologies for detecting mutations and CRISPR gene editing tools are some technical developments. These advancements have led to innovations and ensured availability of advanced cancer gene therapies driving the industry trends.

Request for a sample copy of this report @https://www.decresearch.com/request-sample/detail/763

Estimates have stated that the global cancer gene therapy market value is likely to cross an annual valuation of USD 2.5 billion by 2025.

Technological developments are majorly fueling cancer gene therapy growth. Genetically modified genes which block tumor growth have been incorporated into recent cancer therapies. Efficient isothermal amplification techniques and PCR technology are transforming the way gene mutations are detected. Recent launch of CRISPR gene editing tools is claimed to help enhance the process of gene therapy development. These innovations and advancements in technology are anticipated to propel cancer gene therapy industry size.

The in-vivo segment is estimated to witness about 22% growth over the forecast period owing to its multiple offered benefits. In-vivo gene therapy consists of direct delivery of therapeutic genes into the target cell, a process which has shown effective results in cancer treatment. Viral vectors are delivered using in-vivo gene therapy which help in stopping the activity of tumor inducing genes and has exhibited positive results in clinical trials.

Biopharmaceutical firms held approximately 48% of the industry revenue share in 2018. Major firms such as Roche and Novartis are working on cancer gene therapies that have high adoption rate of both non-viral and viral vectors. These firms are also carrying out clinical trials that are favoring the demand for such vectors, consequently driving market growth.

Broadening awareness regarding the availability of advanced cancer therapies have stimulated the market for cancer gene therapy in China. Government initiatives and funds have encouraged researchers to carrying out extensive R&D activities linked to cancer gene therapy.

Meanwhile, on a global landscape, companies like Vigene Biosciences, Cobra, Uniqure, Sirion Biotech, Bluebird Bio, Caribou, Ziopharm, Finvector, Cellectis and Sarepta Therapeutics are leading the cancer gene therapy market. These firms are focusing on enhancing their market position through business strategies such as product launches, mergers and acquisitions, among others.

Browse full table of contents (TOC) of this report @ https://www.decresearch.com/toc/detail/cancer-gene-therapy-market

Partial Chapter of the Table of Content

Chapter 4. Cancer Gene Therapy Market, By Type

4.1. Key segment trends

4.2. Ex-vivo

4.2.1. Market size, by region, 2014 2025 (USD Million)

4.3. In-vivo

4.3.1. Market size, by region, 2014 2025 (USD Million)

Chapter 5. Cancer Gene Therapy Market, By Product

5.1. Key segment trends

5.2. Viral vectors

5.2.1. Market size, by region, 2014 2025 (USD Million)

5.2.2. Adenoviruses

5.2.2.1. Market size, by region, 2014 2025 (USD Million)

5.2.3. Lentiviruses

5.2.3.1. Market size, by region, 2014 2025 (USD Million)

5.2.4. Retrovirus

5.2.4.1. Market size, by region, 2014 2025 (USD Million)

5.2.5. Adeno associated virus

5.2.5.1. Market size, by region, 2014 2025 (USD Million)

5.2.6. Herpes simplex virus

5.2.6.1. Market size, by region, 2014 2025 (USD Million)

5.2.7. Vaccinia virus

5.2.7.1. Market size, by region, 2014 2025 (USD Million)

5.2.8. Others

5.2.8.1. Market size, by region, 2014 2025 (USD Million)

5.3. Non-viral vectors

5.3.1. Market size, by region, 2014 2025 (USD Million)

5.4. Others

5.4.1. Market size, by region, 2014 2025 (USD Million)

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Cancer Gene Therapy Market 2020 by industry trends, statistics, key companies growth and regional forecast - News by Decresearch