BioPharma Reporter

APRIL 22, 2024

SK pharmteco has signed an agreement with Ferring Pharmaceuticals, to scale up commercial manufacturing capacity for the drug substance of Ferringâs intravesical gene therapy Adstiladrin.

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. Here’s what this means for pharmaceutical leaders and patients.

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STAT News

JULY 29, 2022

Sarepta Therapeutics said Friday that it intends seek regulators’ approval for its gene therapy to treat Duchenne muscular dystrophy earlier than expected — a risky move but one that could help the company reach the market much faster. In early Friday trading, Sarepta shares rose 12% to $96.50.

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pharmaphorum

JANUARY 12, 2022

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. point increase on the NSAA scale with the gene therapy, while the control group saw a 0.7-point

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BioPharma Reporter

FEBRUARY 21, 2023

Three months after receiving the US green light, CSLâs candidate Hemgenix has become the first gene therapy to gain EU approval for the treatment of hemophilia B.

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BioPharma Reporter

MARCH 19, 2024

Food and Drug Administration has cleared AGC Biologicsâ Milan site to begin manufacturing of Orchard Therapeuticsâ Lenmeldy (atidarsagene autotemcel), a gene therapy for early-onset metachromatic leukodystrophy (MLD).

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BioPharma Reporter

AUGUST 18, 2022

Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

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BioPharma Reporter

JANUARY 10, 2023

Catalent is to support the manufacture of Sarepta Therapeutics' gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD).

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BioPharma Reporter

JANUARY 31, 2023

UK based, Aptamer Group, and Hong Kong headquartered, BaseCure Therapeutics, are paring up on the development of Optimer-targeted gene therapies.

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BioPharma Reporter

APRIL 24, 2023

bluebird bio has announced the submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD).

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BioPharma Reporter

JUNE 29, 2023

Astraveus, the creator of modular, microfluidic cell foundries for cell and gene therapy (CGT) manufacturing, has raised â16.5 million in series seed financing.

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BioPharma Reporter

MARCH 2, 2023

Spark Therapeutics, a Roche company, has officially started work on its US$575m gene therapy innovation center in West Philadelphia. The center is expected to be completed by 2026.

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BioPharma Reporter

FEBRUARY 27, 2023

Chinese companies Porton Advanced Solutions and Yinjia Biosciences have signed a pact aimed at supporting further development of cell and gene therapies.

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Drug Discovery World

MARCH 16, 2023

The cell and gene therapy (CGT) market is rapidly expanding, and projections suggest it will reach over $90B by 2023. Challenges of cell and gene therapy manufacturing Compared to traditional biologics, CGTs are living drugs, making their development and manufacturing substantially more complex.

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BioPharma Reporter

MARCH 6, 2023

Sarepta Therapeutics would seem to be edging closer to accelerated FDA approval of its gene therapy for Duchenne muscular dystrophy (DMD).

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BioPharma Reporter

OCTOBER 31, 2023

The London and New York-based gene therapy player MeiraGTx Holdings has received a $30 million investment from Sanofi in addition to potential strategic deals with the big pharma company down the road.

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Drug Discovery World

NOVEMBER 24, 2022

DDW’s Diana Spencer asks what will be needed for Europe to compete in the growing global market for advanced therapies. . Given the long-term focus on investment decisions, the report states that regions that offer long-term stable environments coupled with growing markets will benefit from decreased perceived risk.

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BioPharma Reporter

APRIL 6, 2022

The FDA has released two draft guidance documents: for human gene therapy products incorporating human genome editing, and for CAR T cell products.

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BioPharma Reporter

DECEMBER 22, 2021

There is a healthy appetite for continued investment in gene therapy, in new modalities and more indications, says an industry insider as he reflected on the prospects for the US gene therapy sector next year.

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pharmaphorum

NOVEMBER 20, 2020

The inaugural Gene Therapy Medical Affairs Summit is dedicated to exploring the pivotal internal and external role that Medical Affairs plays in delivering gene therapies to patients. The post Gene Therapy Medical Affairs appeared first on. Access the official agenda for more information.

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BioPharma Reporter

JULY 26, 2022

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, is teaming up with Roche to develop novel AAV gene therapy vectors for eye diseases.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

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BioPharma Reporter

SEPTEMBER 23, 2021

This month sees the launch of a new gene therapy player, Opus Genetics, a company backed and spun out by leading patient group Foundation Fighting Blindnessâ venture arm, the Retinal Degeneration Fund (RD Fund).

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BioPharma Reporter

DECEMBER 22, 2021

Novartis will acquire UK-based Gyroscope Therapeutics, the company behind a one-time investigational gene therapy for geographic atrophy (GA).

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Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? This will give us the opportunity to open dialogues with the regulatory body and gain invaluable experience in the marketing of our CGT product.”

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BioPharma Reporter

SEPTEMBER 13, 2021

AbbVie is aligning with Regenxbio to develop and commercialize a potential one-time gene therapy for the treatment of chronic retinal diseases.

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BioPharma Reporter

MARCH 25, 2022

Exothera, a CDMO specializing in the industrialization of vaccine and gene therapy processes, reports that it has achieved GMP certification for its facilities in Jumet, Belgium.

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Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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pharmaphorum

NOVEMBER 8, 2021

French drugmaker Sanofi has made a $40 million investment in Gyroscope Therapeutics, with another $20 million potentially on offer as the UK biotech advances its clinical-stage gene therapies for ophthalmic diseases. CFI regulates the activity of the complement immune system which is over-activated in GA.

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BioPharma Reporter

SEPTEMBER 3, 2020

AskBio, which develops adeno-associated virus (AAV) gene therapies for genetic disorders, has won an R&D grant valued at Â2m (around US$2.7m) from Scottish Enterprise.

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FDA Law Blog

MARCH 31, 2024

Gibbs — On March 21, 2024, the House Energy and Commerce held a subcommittee hearing titled “Evaluating Approaches to Diagnostic Test Regulation and the Impact of the FDA’s Proposed Rule.” By Ana Loloei & Jeffrey N. FDA, which was not invited to participate, would surely have concurred. These are areas where “revenue is modest,” she said.

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BioPharma Reporter

JUNE 21, 2022

The developer says it is harnessing parvovirus vectors that can deliver larger gene therapy payloads with enhanced tissue specificity and with minimal neutralizing immunity.

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BioPharma Reporter

MARCH 30, 2023

Pharmaron Beijing Co Ltd says its Liverpool, UK based gene therapy CDMO has obtained a grant from the UK governmentâs life sciences innovation manufacturing fund (LSMIF) to expand its facilities.

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BioPharma Reporter

APRIL 29, 2021

Adverum Biotechnologies has reported a setback in its trial exploring gene therapy for the treatment of diabetic macular edema (DME).

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BioPharma Reporter

JANUARY 16, 2023

The European Medicines Agency (EMA) is planning to alert healthcare professions about liver failure cases linked to Zolgensma, a gene therapy against spinal muscular atrophy (SMA), which is developed by Novartis.

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BioPharma Reporter

MARCH 13, 2023

The extremely high costs of gene therapies are unsustainable, and a global commitment to affordable, equitable access to these treatments is urgently needed, concluded the organising committee of a conference on human genome editing.

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