Gene therapy
BioMarin Pharmaceuticals on Monday said it is restricting sales of its hemophilia A gene therapy to three countries in an effort to reduce costs and help the treatment become profitable by 2025.
Longeveron and Lexeo Therapeutics are working on CGT therapies to treat Alzheimer’s disease, but it’s not clear whether they have a better chance of success than traditional approaches.
Sarepta has been hit with another patent infringement lawsuit, this time from Sanofi and its subsidiary Genzyme alleging that the biotech used protected technology related to AAV vectors.
The advantages of using circular RNAs—including increased durability, enhanced protein expression and substantially lower manufacturing costs compared to linear mRNAs—have driven a spate of investment in this technology.
Fatalities are an unfortunate reality of clinical trials. How can companies best protect themselves?
The company’s candidate, giroctocogene fitelparvovec, met its primary and key secondary objectives of superiority compared to the standard treatment of regular Factor VIII infusions.
The Department of Health and Human Services’ Office of the Inspector General found that bluebird bio’s fertility support program for its gene therapies could potentially violate federal anti-kickback statutes.
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.
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