Research Reversal of Parkinson's in Mice, Safeguarding Primates from HIV, Leading Biotech Hubs, and Sarepta

In a recent development, Sarepta Therapeutics is grappling with a regulatory conflict surrounding its DMD gene therapy, Elevidys. This conflict has led to a temporary pause in shipments of the therapy. The news comes after a series of patient deaths related to Sarepta's gene therapies, resulting in a significant restructuring that saw the company cut 500 jobs, representing 36% of its workforce.

The regulatory conflict has been a topic of discussion, with the Food and Drug Administration (FDA) playing a key role. Vinay Prasad, MD, who was instrumental in the agency's brief get-tough approach towards Sarepta, has departed. However, Prasad resumed his role as the director of the FDA's Center for Biologics Evaluation and Research (CBER) less than two weeks after his resignation, taking the reins once again.

Meanwhile, another biotech company, Sarepta's rival, is making headlines. A single gene therapy injected at birth offers lasting protection from HIV in infant rhesus macaques, according to a study published in the journal GEN. This breakthrough could potentially revolutionise the fight against HIV.

Elsewhere in the biotech world, a GLP-1 drug named Semaglutide has been linked to reduced muscle strength in mice. This revelation could have significant implications for the development and use of this drug.

In a positive note, a study published in GEN shows that breathing low oxygen can restore neuron function in a mouse model of Parkinson's disease. This could pave the way for new treatments for the debilitating condition.

The biotech industry is also looking forward to key factors shaping this year's A-List of Top 10 U.S. Biopharma Clusters. Registration for The State of Biotech Summit is available for those interested in learning more about the industry's latest developments.

Finally, the Behind the Breakthroughs podcast, hosted by Jonathan D. Grinstein, PhD, and the Touching Base Podcast, hosted by Corinna Singleman, PhD, are worth a listen for insights into the latest in biotech and pharmaceuticals.

Stay tuned for more updates on these stories and more on GEN.

Research Reversal of Parkinson's in Mice, Safeguarding Primates from HIV, Leading Biotech Hubs, and Sarepta