pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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Gene Therapy Gene Trials FDA Approval 111

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

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Gene Therapy Gene Medicine Trials 98

FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Gene Therapy Gene FDA Approval Production 119

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. How Can Study Protocols Be More Effective?

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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

FEBRUARY 9, 2023

ReviR Therapeutics has signed a research collaboration and option-to-license agreement with Asieris Pharmaceuticals to discover new oncology therapeutics. Its initial focus is to develop therapeutics in oncology, CNS, and genetically defined disease indications.

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FDA Law Blog

MARCH 13, 2023

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials.

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Worldwide Clinical Trials

NOVEMBER 27, 2023

Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023.

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Gene Editing Gene Gene Therapy In-Vivo 189

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The option licensing agreement is based on early-stage clinical results.

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Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein.

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Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more. 1] BioNews. Hemophilia News Today. Biores Open Access.

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Genomics Genome Medicine Gene Therapy 244

pharmaphorum

OCTOBER 26, 2020

Bayer is making clear its ambitions in cell and gene therapy with a $4 billion acquisition of US biotech Asklepios BioPharmaceutical, also known as AskBio. Cell and gene therapy is viewed as one of the most promising areas in pharma, with several biotech and big pharma firms making plays in the area.

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Gene Therapy Gene Genetic Disease Genetics 59

Pharmaceutical Technology

APRIL 6, 2023

Ginkgo Bioworks has strengthened its end-to-end R&D capabilities in gene therapy with the purchase of adeno-associated virus (AAV) capsid discovery and engineering assets from StrideBio, for an undisclosed sum. The new capabilities and IP will be incorporated under Ginkgo’s end-to-end AAV gene therapy development platform.

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Pharmaceutical Technology

AUGUST 24, 2022

Lacerta Therapeutics has entered a new licensing and research partnership agreement with Eli Lilly and Company subsidiary, Prevail Therapeutics, to discover and develop adeno-associated virus (AAV) capsids for treating central nervous system (CNS) diseases. .

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pharmaphorum

OCTOBER 18, 2022

Eli Lilly has agreed to buy hearing loss gene therapy developer Akouos in a deal that could value the company at around $610 million – if its lead candidate progresses as hoped in the clinic. The buyout – for $12.50 The buyout – for $12.50 per month if conditions are not met, until the end of 2028 when they will all expire.

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Gene Therapy Gene Genetics FDA Approval 52

STAT News

MARCH 9, 2023

In the spring of 2013, I had just visited a lab focused on gene and cell therapies at Baylor College of Medicine. Bluebird Bio , which would soon launch a successful IPO , had licensed its technology from the same lab and was about to announce a major collaboration with Celgene and Baylor. Read the rest…

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Gene Gene Therapy Licensing Licensing 98

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

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pharmaphorum

OCTOBER 29, 2020

Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7

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pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. Translarna was the first licensed treatment for DMD which addresses the loss of dystrophin. Translarna was the first licensed treatment for DMD which addresses the loss of dystrophin.

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Gene Therapy Gene FDA Approval DNA 98

The Pharma Data

JANUARY 3, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that U.S. GM1 is a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available. PHILADELPHIA, Jan. About PBGM01.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

Delveinsight

JANUARY 12, 2021

Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant. Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences.

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Licensing Licensing Antibody Gene Therapy 52

Delveinsight

DECEMBER 8, 2020

KaliVir, Astellas Pharma forms a licensing deal for VET2-L2 oncolytic virus. KaliVir Immunotherapeutics and Astellas Pharma entered into a worldwide exclusive licensing agreement for the development, research, and commercialization of VET2-L2 to widen the horizon of therapeutic approaches available in the Immuno-Oncology market.

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Licensing Licensing Gene Therapy Antibody 52

The Pharma Data

JANUARY 27, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. The gene therapy utilizes an AAV1 viral vector to deliver a modified DNA encoding the GRN gene to a patient’s cells.

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Delveinsight

DECEMBER 17, 2020

Atsena raises USD 55 Million financings for blindness gene therapy. University of Florida (UF) spinout Atsena Therapeutics has completed a USD 55 million financing that will benefit its gene therapy for a common cause of blindness in children into pivotal trials. million in financing.

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Gene Therapy Gene DNA Genetics 96

Delveinsight

DECEMBER 3, 2020

Janssen buys a gene therapy asset. Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences. There are presently no medical treatments, which can regenerate retinal cells that have atrophied, though gene therapies and stem cell therapies are the leading R&D hopes in this arena.

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Gene Therapy Gene Antibody Research 52

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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XTalks

MARCH 21, 2023

Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall Rett syndrome is typically caused by a genetic mutation on the MECP2 gene. Significant development regression associated with the disease occurs after a period of normal development from six to 18 months of age.

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FDA Approval Gene Therapy Gene Genetics 94

pharmaphorum

JUNE 7, 2022

million licensing agreement with Alectos Therapeutic, as it continues to rebuild a pipeline behind its side-lined Alzheimer’s therapy Aduhelm. It’s not the first time that Biogen has licensed rights to a drug targeting lysosomal function in Parkinson’s disease.

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pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. The post Bayer trumpets $1bn CRISPR deal with Mammoth Bio appeared first on.

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pharmaphorum

DECEMBER 9, 2022

This means that enrolment is now completed for the initial target or morphological cohort to form the basis of a Biological License Application (BLA) submission by the end of CY2023 to the US Food and Drug Administration (FDA). Syncona has agreed to invest circa $28 million (£22.9 The tender offer for that acquisition was approximately $23.5

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The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

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The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

Delveinsight

AUGUST 5, 2021

Sanofi is betting the genetic technology behind the fast development of two highly effective coronavirus shots last year will result in vaccines for other viruses as well as drugs for diseases of the lung and liver, announcing a deal to acquire a research partner Translate Bio for USD 3.2 Sanofi bets USD 3.2 No financial terms were revealed.

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Vaccine Vaccination Gene Therapy Development 98

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

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Pharmaceutical Technology

MAY 26, 2023

Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines. As per the company, dbDNA is suitable for rapid, scalable manufacture of GMP DNA and can incorporate gene sequences of sizes ranging from 500bp to 20kb, which are typically unstable as pDNA.

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XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. It may also allow for priority or rolling review of a company’s Biologics License Application (BLA).

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