Phase II
The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.
Johnson & Johnson and Addex Therapeutics’ epilepsy hopeful ADX71149 did not meet its primary endpoint of time to reach baseline seizure count in a mid-stage trial, as the partners look to determine the next steps for the program.
A one-fifth dose of Bavarian Nordic’s mpox vaccine Jynneos can elicit a similar antibody response as its standard-dose regimen, according to findings presented Saturday at the European Society of Clinical Microbiology and Infectious Diseases Global Congress.
With GLP-1 agonists slated to become the best-selling drugs in 2024, the biopharma industry is already pivoting to explore treatments that preserve muscle mass as patients shed pounds.
Several companies—including Roche, Curemark and Yamo Pharmaceuticals—are running clinical trials of treatments that aim to offset the myriad challenges of autism.
While the San Diego-based biotech awaits a Phase IIa data readout for its lead lung disease candidate, Endeavor BioMedicines’ next-generation HER3-targeted antibody-drug conjugate will start a Phase I/II trial later this year.
An undisclosed once-daily oral dose of Neurocrine Biosciences’ Takeda-partnered investigational drug significantly reduced symptom severity in adults with major depressive disorder.
As the Phase III amyotrophic lateral sclerosis pipeline thins out, the ALS community is placing its hopes on earlier-stage trials sponsored by Denali Therapeutics, PTC Therapeutics and more.
Phase II data indicate Larimar Therapeutics’ injectable therapy nomlabofusp could go head-to-head in the market with Biogen’s Skyclarys.
Drugmakers, including the genetic testing company 23andMe, are using novel approaches to stimulate NK cells’ intrinsic ability to recognize and kill tumor cells.
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