Gene therapy
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.
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.
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