Sarepta Therapeutics, Inc.
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Researchers at the University of Texas Southwestern Medical Center used CRISPR gene editing to treat Duchenne muscular dystrophy (DMD) in dogs. Their work was published in the journal Science.
Pfizer terminated two clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD).
Recently, Milton Packer, a renowned cardiologist at Baylor Scott & White Health, wrote a criticism of the Orphan Drug Act and the resulting problems caused by the biopharma industry. John LaMattina, formerly president of Pfizer Global Research and Development and a current senior partner at PureTech Ventures, responded to the editorial in Forbes. Let’s take a look.
It’s not easy to predict trends in drugs, especially with breakthroughs in immunology and genetic engineering often causing dramatic changes in how biopharma companies approach new drugs.
Cambridge, Mass.-based Sarepta Therapeutics expanded its gene therapy pipeline up to 11 in an equity deal with Lacerta Therapeutics. Investing $30 million into Alachua, Florida-based Lacerta Therapeutics, Sarepta gained access to the company’s AAV-based CNS-targeted gene therapies.
This week, the Business Journals compiled a list of the 25 companies across the United States with the highest median salary for employees. According to the list, median salaries for these companies ranked from $198,000 to $495,000. Of those companies listed, more than half were in the pharma or biotech industries.
The FDA notified Sarepta Therapeutics that its Phase I/IIa Micro-Dystrophin Gene Therapy Trial for Duchenne muscular dystrophy (DMD) has been placed on a clinical hold because of contamination of its therapeutic.
Biotech manufacturing is the non-glamorous side of biopharma, although its impact on the economy shouldn’t be underestimated. Here’s everything you need to know.
Miracles, it seems, have a high price tag. At least, if those miracles are miracle drugs. There’s no doubt that trends in gene therapy and immuno-oncology are producing drugs that are as close to miraculous as we’re likely to get, doing a great job, generally, in beating back diseases that to this point were untreatable or didn’t respond well to other therapies.
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