Drug Development
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.
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Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated virus gene therapies to more rare disease patients.
Amylyx’s recent decision to withdraw its ALS drug Relyvrio from the market highlights an important business decision for companies: when to continue marketing or investigating a drug that has failed a pivotal or confirmatory study.
As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.
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Eisai presented a plethora of data on the drug at the Alzheimer’s Association International Conference, including a study showing the consequences of pausing treatment.
The FDA will have six months to review Vertex Pharmaceuticals’ suzetrigine, potentially the first new class of drugs for acute pain in more than 20 years, according to the company.
Second-quarter earnings season continues with Big Pharma beating Wall Street expectations, the author of an encrypted email sent to BioSpace has a proposal for Moderna and Merck, Roche and Viking seek quicker entry to the obesity market, and AAIC is in full swing.
Sangamo and Pfizer’s hemophilia A gene therapy candidate scored a Phase III victory last week. However, with the genomic medicine company soon to run out of cash, Sangamo’s short-term prospects look bleak but not unsalvageable, analysts say.
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.
Cognitive function in the liraglutide cohort declined 18% slower than in the placebo arm over one year of treatment, researchers announced Tuesday at the Alzheimer’s Association International Conference.
BioNTech and Regeneron will face off against Merck and Moderna, which are advancing their investigational cancer vaccine mRNA-4157/V940 in combination with Keytruda, in advanced melanoma.
Adding to growing evidence of its benefits beyond glucose control and weight loss, Novo Nordisk’s Ozempic could also potentially help diabetics quit smoking, according to new research.
AstraZeneca is seeking a fixed-duration approval for Calquence, which will allow patients with chronic lymphocytic leukemia to take breaks from the therapy and prevent excessive toxicities and drug resistance.
Back-to-back failures in psoriasis and Crohn’s disease have forced Ventyx Biosciences to abandon the development of its investigational oral TYK2 inhibitor VTX958.