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FDA approves first gene therapy for adults with haemophilia B

CSL’s Hemgenix allowed patients to produce mean factor IX activity of 39% at six months

- PMLiVE

Sarepta to assess imlifidase as pre-treatment to Duchenne muscular dystrophy gene therapy

The company submitted a Biologics License Application to the FDA for SRP-9001 in September

Cell and gene therapies as depicted via a DNA strand

The fast-paced future of cell and gene therapies

In this report, we interview patients, researchers, and biopharmaceutical leaders to explore the complex landscape for the development of cell and gene therapies and share commercial recommendations from our specialist...

Avalere Health

Biomarin

BioMarin’s application for haemophilia A gene therapy accepted by FDA

If approved, the treatment would be the first gene therapy in the US for this indication

- PMLiVE

The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia

With transformative treatments that leverage CRISPR technology, there are many challenges to overcome throughout the journey of development through to patient access. We interviewed a researcher at a top 20...

Avalere Health

- PMLiVE

Pfizer and Sangamo Therapeutics reopen recruitment for haemophilia A gene therapy trial

The trial was voluntarily paused by the two partners last year and later placed on hold by the FDA

- PMLiVE

EC approves BioMarin’s gene therapy Roctavian for severe haemophilia A

Approximately 20,000 adults are affected by haemophilia A across more than 70 countries in Europe, the Middle East and Africa

- PMLiVE

Sarepta to submit application for Duchenne muscular dystrophy gene therapy

The condition occurs in approximately one in every 3,500-5,000 newborn males worldwide

- PMLiVE

bluebird bio maintains its focus on gene therapy opportunities during structural changes

The company plans to reduce its workforce amid efforts to reduce its operating costs

- PMLiVE

Novartis and Voyager Therapeutics agree on gene therapy deal worth 1.7bn

The licence option agreement is for next-generation gene therapy vectors for neurological diseases

- PMLiVE

bluebird bio shares therapy updates for 2022

The company provided information for its first two gene therapies and its lovo-cel BLA submission for sickle cell disease

Takeda agrees on $2bn deal with Code Bio to expand its gene therapy programmes

The pharma company will gain opt-in rights for four rare diseases candidates

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