Here are the latest stories being discussed in biopharma today:
**Story 1: mRNA Search-and-Replace Editing Emerges**
Biotech companies and academic institutions are developing an innovative technique similar to DNA-editing. Known as RNA writing, this technique allows the replacement of unwanted mRNA sequences, offering a potentially safer and more efficient alternative. The RNA writing technology may provide crucial treatments for diseases with multiple varied gene errors, too substantial for existing gene therapy methods. Upcoming clinical trials will determine the efficacy of these techniques in various diseases.
**Story 2: Gilead Acquires CymaBay for $4.3B**
Gilead Sciences has announced plans to acquire biotech company CymaBay Therapeutics for $4.3 billion in cash. The purchase comes ahead of CymaBay’s FDA approval decision in August 2022 for seletalisib, a drug candidate for Mycobacterium avium complex (MAC) lung disease. This disease mainly affects at-risk groups like postmenopausal women and those aged over 65.
**Story 3: Late-Stage Trial Halted by Infectious Disease Biotech**
California-based biotech company AN2 Therapeutics has voluntarily halted a late-stage clinical trial due to potential lower-than-expected efficacy. The Phase II/III trial was testing its lead drug for Mycobacterium avium complex (MAC) lung disease. Its stock price plummeted by up to 73%, and the company is currently awaiting recommendations for next steps from the Data and Safety Monitoring Board.
**Story 4: Larimar Releases Key Biomarker Data for Friedreich’s Ataxia Drug**
Larimar Therapeutics announced new findings demonstrating that its Friedreich’s ataxia treatment, nomlabofusp, effectively increased levels of a key protein called frataxin in a mid-stage study. The company has been in discussions with the FDA about potentially using the frataxin biomarker data for an accelerated approval submission in 2025.
**Story 5: Gilead to Snap up CymaBay Therapeutics for $4.3B**
Gilead Sciences will acquire CymaBay Therapeutics for $4.3 billion in cash. The deal comes as the FDA is set to make an approval decision on CymaBay Therapeutics’ rare liver disease drug candidate, seletalisib, for primary biliary ch