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Sarepta Therapeutics

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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

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Gilead’s Trodelvy granted FDA priority review for metastatic breast cancer

Phase 2 study results showed a 34% reduction in risk of disease progression or death

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GSK’s Boostrix approved by FDA for use during pregnancy to prevent whooping cough in infants

4.2% of the cases of pertussis reported in the US in 2021 were in infants younger than six months

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FDA approves scPharmaceuticals’ on-body infusor for congestive heart failure

The at-home device delivers the equivalent of what a patient would receive by way of IV

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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

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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

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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

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FDA lifts hold on Gilead’s clinical trials for leukaemia

Following a review of the safety data, enrolment can be resumed for the MDS and AML clinical trials

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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

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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

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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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