Clinical research
Although it was a relatively slow week for clinical trial updates, there were still a number of announcements. Here’s a look.
Although CRISPR gene editing is touted as likely to revolutionize medicine, the actual proof of its effectiveness and safety in treating diseases has been slow in coming. At least until now. Sort of.
Emeryville, California-based Zogenix announced positive topline data from its Phase III trial of Fintepla (fenfluramine) in Lennox-Gastaut Syndrome (LGS).
Biogen successfully fended off a patent challenge from generic company Mylan over its multiple sclerosis (MS) drug Tecfidera.
Genentech’s Risdiplam showed significant improvement in motor function in people aged 2-25 who have been diagnosed with Type 2 or 3 spinal muscular atrophy.
The immuno-oncology therapy, CAR-T, utilizes specific types of immune cells, T-cells, which are drawn from the cancer patient, supercharged, and infused back into the patient. Now, The University of Texas MD Anderson Cancer Center has developed a slightly different approach using a different type of immune cell called Natural Killer (NK) cells.
Boston is getting a new tenant focused on rare diseases. Italian pharma company Chiesi Farmaceutici S.p.A is launching a U.S.-based subsidiary Chiesi Global Rare Diseases with a focus on advancing research and new product development for rare and ultra-rare diseases.
The National Institutes of Health ceased administration of an investigational HIV vaccine after interim data revealed that the treatment was not working in preventing the virus.
Evotec SE announced that its partner Bayer AG has decided to advance a further programme from the endometriosis multi-target alliance into clinical Phase I development.
The company plans to present detailed study results from the WILLOW study at an upcoming medical meeting.
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