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Sickle cell pipeline narrows as gene therapy developers rethink research plans

Bio Pharma Dive

Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.

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Research consortium picks eight rare diseases to advance gene therapies

Pharmaceutical Technology

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.

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Patient dies in Pfizer study of Duchenne gene therapy

Bio Pharma Dive

The company is working with trial researchers to investigate further. Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly.

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Voyager licenses gene therapy tools to Novartis

Bio Pharma Dive

The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.

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5 questions facing gene therapy in 2022

Bio Pharma Dive

As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines.

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Magazine: What’s next for AAV gene therapies in 2024?

Pharmaceutical Technology

In this issue: High-profile gene therapies that use AAV vectors, new research into HIV-TB coinfection treatments, traceability technologies for the supply chain, and more.

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Forge Biologics and Labcorp partner for gene therapies development

Pharmaceutical Technology

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.