Limb-girdle muscular dystrophy
Lilly and MeiraGTx partner in $475m ophthalmology deal
The companies will develop and commercialise therapies for genetic conditions
The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter
By Sarah Mohamad and Lauren Coleman-Bennett
CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix
The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US
NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease
About 15,000 people in England are living with the inherited blood disorder
Vertex and Orum partner on gene editing conditioning drugs in deal worth $945m
The agreement gives Vertex the rights to use Orum’s targeted protein degradation technology
FDA expands indication for Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys
The neuromuscular condition is estimated to affect one in every 3,500 male births worldwide
FDA publishes final industry guidance for CAR-T and gene therapies
The new guidance can also be applied to other genetically modified lymphocyte products, including CAR NK cells
AstraZeneca and Cellectis enter gene therapy partnership worth over $2.2bn
The deal gives AZ access to Cellectis’ gene editing technologies and manufacturing capabilities
BioMarin announces first patient dosed with haemophilia A gene therapy in Europe
The therapy was granted conditional marketing authorisation by the EC in August 2022
Regeneron to acquire Decibel Therapeutics in deal worth up to $213m
The transaction expands the company’s gene therapy and hearing loss pipeline
NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy
The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors