Spark Therapeutics
NEWS
Gene therapy has been a dream for many researchers for decades—inserting a healthier gene into patients’ cells to replace defective genes and curing diseases. One disease that would seem to be a good target for gene therapy is hemophilia.
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.
An investigational gene therapy treatment for hemophilia A has seen a 97 percent response rate after one treatment for the 12 trial participants, Spark Therapeutics announced this morning.
Akouos, based in Boston, announced a Series A financing worth $50 million. The round was led by seed investors 5AM Ventures and New Enterprise Associates (NEA).
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.
It feels like we’re standing on the edge of a precipice. Despite the ho-hum recent performance of biotech stocks, amazing things are about to happen. And one of the most amazing of all is gene therapy.
Axovant Sciences, one of Vivek Ramaswamy’s biotech startups, is taking another shot, this time at Parkinson’s disease.
Shares of CRISPR Therapeutics plunged dramatically Wednesday after the U.S. Food and Drug Administration placed a clinical hold on a planned sickle cell disease treatment the company was in the process of developing with Vertex Pharmaceuticals.
Vyriad, based in Rochester, Minnesota, announced it is building out its facility in 25,000 square feet of space it is leasing on the IBM Campus in Rochester.
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