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Category: Cell and Gene Therapy


Cell and Gene Therapy, Industry

BioMarin’s hemophilia gene therapy recommended for approval in Europe

June 24, 2022

Via: Biopharma Dive

The regulatory agency that recommends whether new drugs should be approved in Europe has thrown its support behind a gene therapy for the rare bleeding disorder hemophilia A, putting the potentially curative treatment one step closer to market. People with […]


Cell and Gene Therapy, Industry

A biotech startup launches with $38M to develop a cystic fibrosis gene therapy

June 21, 2022

Via: Biopharma Dive

Vertex’s latest treatment for cystic fibrosis is a “triple combination” drug called Trikafta, which like others from Vertex boosts the activity of a protein that’s defective in people with the disease. It has quickly become a go-to treatment, entrenching Vertex’s […]


Cell and Gene Therapy, Industry

Coave Therapeutics and ABL Enter Gene Therapy Collaboration

June 13, 2022

Via: Contract Pharma

Coave Therapeutics, a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases, and ABL, a pure play contract development and manufacturing organization (CDMO) specialized in the development and manufacturing of viruses for […]


Cell and Gene Therapy, Industry

FDA panel backs Bluebird gene therapy despite safety risks

June 9, 2022

Via: Biopharma Dive

An experimental gene therapy could benefit young boys with a rare genetic disease, despite considerable concerns that treatment may cause cancer, a panel of Food and Drug Administration advisers unanimously agreed in a high-profile meeting Thursday. The therapy, an infusion […]


Cell and Gene Therapy, Industry, Manufacturing, Research and Development

Regenxbio Opens Gene Therapy Manufacturing Facility

June 9, 2022

Via: Contract Pharma

Regenxbio recently celebrated the opening of its new Manufacturing Innovation Center gene therapy manufacturing facility. “Launching operations at our Manufacturing Innovation Center is an important milestone in the evolution of REGENXBIO,” said Kenneth T. Mills, president and CEO of REGENXBIO. […]


Cell and Gene Therapy, Industry

Immatics and Editas join up to bring CRISPR to ‘gamma delta’ cell therapy

June 7, 2022

Via: Biopharma Dive

Though small in scope, Immatics and Editas’ deal adds to a flurry of recent activity involving treatments that harness gamma delta T cells, rare white blood cells with unique tumor-fighting capabilities. Unlike the T cells used in Novartis, Gilead and […]


Cell and Gene Therapy, Industry

BioMarin’s hemophilia A gene therapy further delayed amid new FDA questions

June 1, 2022

Via: FierceBiotech

BioMarin’s commercial aspirations for its hemophilia gene therapy remain on ice after the FDA lobbed additional questions for the company to address, pushing back an expected approval submission to September. BioMarin was vague on what the FDA is asking for, […]


Cell and Gene Therapy, Industry

BioMarin delays planned FDA filing for hemophilia gene therapy

May 31, 2022

Via: Biopharma Dive

BioMarin’s road back to the FDA keeps getting longer, with the agency’s request adding new uncertainty to a resubmission that’s been in the works for nearly two years. The California biotech disclosed the FDA’s feedback alongside updated five- and six-year […]


Cell and Gene Therapy, Industry

Evotec Adds Cell Therapy Mfg. Facility with Rigenerand Acquisition

May 31, 2022

Via: Contract Pharma

Despite a long history of difficulties in this area, FDA once again is taking steps to facilitate the import of less costly prescription drugs from Canada by states, Indian Tribes, and other entities. The Canadian government has objected to the […]


Cell and Gene Therapy, Industry

Novel cancer vaccines by Dana-Farber and Stanford look beyond T cells to better fight tumors

May 27, 2022

Via: FierceBiotech

Most cancer immunotherapies primarily rely on T cells to neutralize tumors. But in two new studies, scientists at Dana-Farber Cancer Institute and Stanford University have described novel immuno-oncology approaches, showing that leveraging other components of the immune system may achieve […]


Cell and Gene Therapy, Industry

FDA starts clock on review of CSL, UniQure gene therapy

May 25, 2022

Via: Biopharma Dive

The FDA has approved two gene therapies for inherited diseases: the blindness treatment Luxturna in 2017 and the neuromuscular disease therapy Zolgensma in 2019. Since then, despite a boom in gene therapy research among biotech and pharma companies, safety hurdles […]


Cell and Gene Therapy, Industry

CSL Behring gene therapy makes comeback after hold for FDA priority review

May 24, 2022

Via: FierceBiotech

The FDA has accepted CSL Behring’s priority review application for a hemophilia B gene therapy, potentially fast-tracking the drug after a bumpy ride that included a clinical hold—and later, a lift. If approved, etranacogene dezaparvovec—an AAV-5 gene therapy candidate given […]