ROCKVILLE, Md. — REGENXBIO Inc. (Nasdaq: RGNX) today announced topline results from the Phase I/II/III CAMPSIITE® trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, demonstrating that the pivotal phase of the trial met...
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Takeda Korea said Wednesday that it received approval for Obizur, a treatment for bleeding in adult patients with acquired hemophilia A (AHA), from the Ministry of Food and Drug Safety. AHA is a disease characterized by the development of autoimmune antibodies against blood coagulation factor 8, resulting in bleeding-related complications....
PRINCETON, N.J. — Bristol Myers Squibb Company (NYSE: BMY) announced three regulatory acceptances from the U.S. Food and Drug Administration (FDA) and Japan’s Ministry of Health, Labour and Welfare (MHLW) for Breyanzi® (lisocabtagene maraleucel). In the U.S., the FDA has accepted the company’s two supplemental Biologics License Applications (sBLA) for...
SAN DIEGO, Calif. — Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs, today announced it has completed enrollment in the third cohort of patients in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD. “The completion of our...
SAN DIEGO, Calif. — Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs, today announced positive topline results from the second cohort of patients in its Phase 1b MAD study of RGLS8429 for the treatment of ADPKD. The Phase 1b...
HIV ROME, Italy, NEW YORK and CAMBRIDGE, Mass. – ReiThera Srl, the Ragon Institute of Mass General, MIT, Harvard University, and IAVI are pleased to announce a collaboration to develop a novel HIV vaccine candidate that will be composed of ReiThera’s GRAd vector and HIV T cell epitopes identified by...
Three brothers who have been struck down by a rare disease will have to plead their case for life-saving drugs. Daniel Mathieson, 19, and brothers John, 18, and James, 16, have been told they will be lucky to live beyond their 20th birthdays. They already have to use wheelchairs and...
Geneva, Switzerland – RELIEF THERAPEUTICS Holding AG (SIX: RLF, OTCQB: RLFTF)(“Relief”), a biopharmaceutical company seeking to provide patients therapeutic relief from serious diseases with high unmet need, today announced that it has signed and closed the definitive agreement to acquire all outstanding shares of APR Applied Pharma Research S.A. (“APR”),...
Geneva, Switzerland – RELIEF THERAPEUTICS Holding AG (SIX: RLF, OTCQB: RLFTF)(“Relief”), a biopharmaceutical company with its lead compound RLF-100TM (aviptadil) in advanced clinical development to treat severe COVID-19 patients, today provided an update on the development of ACER-001, a proprietary powder formulation of sodium phenylbutyrate (NaPB) designed to be both...
Acer to receive $1 million payment to obtain exclusivity and a $4 million loan from Relief Companies working toward negotiation and execution of a definitive collaboration and license agreement by June 30, 2021 GENEVA and NEWTON, Mass., Jan. 25, 2021 – Relief Therapeutics Holding AG (SIX: RLF, OTCQB: RLFTF)(“Relief”), a...