Nearly four months after Intellia Therapeutics – along with partner Regeneron Pharmaceuticals – reinvigorated the CRISPR space with the first-ever clinical evidence that CRISPR gene editing inside the body can be safe and effective, the asset in question was granted Orphan Drug Designation by the U.S. Food and Drug Administration.
AstraZeneca said on Aug. 26 a late-stage trial showed the company’s medicine for a rare disease that causes accumulation of copper in the body was three times more successful in helping remove deposits from tissues compared with standard care.
Copenhagen, Denmark-based Orphazyme announced the company’s Phase II/III clinical trial of arimoclomal for inclusion body myositis (IBM) failed to hit primary and secondary endpoints.
Novartis released promising interim Phase II data of iptacopan in C3 glomerulopathy (C3G) at the virtual American Society of Nephrology 2020 Annual Meeting.
Pfizer and Opko Health reported their Phase III clinical trial of somatrogan met the primary endpoint compared to Genotropin (somatropin) for treating children 3 to 18 years of age with growth hormone deficiency (GHD).
London-based Freeline announced the closing of a $120 million extended Series C financing to advance the company’s gene therapy programs, and is considering additional capital fundraising options during 2020.
San Rafael, Calif.-based BioMarin Pharmaceutical announced positive Phase III clinical trial results for the company’s gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.
Pfizer announced plans to buy Therachon, a rare disease biotech company based in Basel, Switzerland.