FDA expands approval of gene therapy for patients with Duchenne Muscular Dystrophy
Elevidys now approved for both ambulatory and non-ambulatory patients aged four and older
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Browsing Tag
gene therapy
Beacon Therapeutics’ AGTC-501 holds potential to become first gene therapy in XLRP space: GlobalData
AGTC-501 is highly anticipated as having the potential to become the first gene therapy specifically indicated for X-linked…
CRISPR-based innovations drive $21 bn in pharma licensing deals over five years: GlobalData
The pharma industry has experienced a notable uptick in licensing agreements for innovator drugs incorporating clustered regularly…
US approves first gene therapy for children with rare genetic disease
Orchard, which was acquired by Japanese pharmaceutical firm Kyowa Kirin for $477.6 million last year, said it will provide details…
EMA seeks to issue guidance on liver damage from Novartis’s gene therapy
The statement follows recent deaths due to liver failure after treatment with the gene therapy, the EMA said
US FDA approves Bluebird Bio’s gene therapy for rare neurological disorder
The approval was largely expected after the drug received unanimous endorsement from a panel of outside advisers to the FDA in…
Pfizer to start US trial of gene therapy as FDA lifts hold
The FDA had put Pfizer's trial request on hold after the death of a patient in another early-stage study of the therapy for…
Novartis and Voyager Therapeutics reach licence option agreement for next-generation gene therapy…
Agreement builds on previous Novartis success using emerging technologies to develop first-in-class gene therapies for…
CPhI and Informa Pharma Intelligence conclude 3rd Biopharma Conclave
The conclave drew insights on biologics, cell and gene therapy, vaccines and biosimilars
TSHA-102 from Taysha gets rare paediatric disease and orphan drug designations for Rett syndrome
Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021