Pharmaceutical Company Sarepta Faces Scrutiny; AstraZeneca Places Large Wager; Artificial Intelligence Challenges Interference with Decision-Making Records (IDRs)
In this week's episode of Touching Base, we bring you some significant updates from the world of health and science.
Firstly, we delve into a heartwarming story about eight babies from the U.K. who were born free of mitochondrial disease. This groundbreaking achievement was made possible through a technique known as pronuclear transfer.
Moving on, we discuss a new therapy for limb-girdle muscular dystrophy. Unfortunately, a patient undergoing this therapy recently passed away, leading to a pause in shipments of Sarepta's FDA-approved treatment for Duchenne muscular dystrophy, Elevidys. Sarepta has temporarily halted shipments, complying with the FDA's actions following multiple patient deaths, and a required Black-Box warning for Elevidys in July 2025. This tragic event has sparked ongoing discussion about Sarepta's response to these tragedies associated with its DMD therapy.
In response to this, Sarepta has undergone a restructuring, terminating 500 employees, representing 36% of its workforce.
On a more positive note, AstraZeneca is making strides in the field. They have committed an additional $50 billion to U.S. manufacturing and R&D projects. Furthermore, they have established a new $300M plant for controlling cell therapy production.
The lab of Nobel Prize winner David Baker, PhD, is also making significant strides in the field of drug design. They are developing AI that designs drugs for previously "undruggable" proteins, a breakthrough that could revolutionise the industry.
Finally, the discussion in this episode of Touching Base also touches upon the production of genome editing cures, moving beyond the case of Baby KJ.
Tune in to Touching Base for more insights into these stories and more.
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