Gene therapy
Mid-stage data for 4D Molecular Therapeutics’ wet AMD drug 4D-150 show improvements in visual acuity, reduced injection rate and encouraging safety data.
As the yearslong litigation over ownership of CRISPR gene editing continues, investors have forged ahead with funding the technology’s development by biopharma.
Vertex has filed a complaint against the Department of Health and Human Services, seeking to make its fertility preservation program available to federally insured patients needing Casgevy treatment.
Lexeo Therapeutics’ investigational gene therapy reduces left ventricular volume and wall thickness in patients with Friedreich’s ataxia, according to a small study.
Following a disappointing readout last year, uniQure on Tuesday posted promising Phase I/II data for its investigational gene therapy AMT-130 and nabbed the first-ever Regenerative Medicine Advanced Therapy designation from the FDA in Huntington’s disease.
The groundwork being done in 2024 is building the foundation for global collaboration in the future.
Rocket Pharmaceuticals’ gene therapy Kresladi has been hit with an FDA Complete Response Letter requesting additional chemistry, manufacturing and controls information to complete its review.
After winning expanded approval for its gene therapy for Duchenne muscular dystrophy, Sarepta’s leadership and analysts see a sizeable commercial opportunity on the horizon.
Exsilio Therapeutics emerged from stealth on Tuesday with a platform that leverages mRNA technology to develop redosable genomic medicines for a range of complex diseases.
While Thursday’s label expansion and traditional approval for the gene therapy is an important milestone, many challenges still face the Duchenne muscular dystrophy community.
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