BioSpace News Archive

Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (“Regulus”), today announced that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the Oppenheimer Rare & Orphan Disease Summit on Friday, May 21, 2021 at 11:35 A.M. ET.

REGENXBIO Inc. (Nasdaq: RGNX) today announced a safety update and additional positive interim data from the ongoing Phase I/II 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

Biogen Inc. (Nasdaq: BIIB) today announced topline results from the Phase 2/3 XIRIUS study of cotoretigene toliparvovec (BIIB112), a gene therapy being investigated as a one-time therapy for patients with X-linked retinitis pigmentosa (XLRP).

Solid Biosciences Inc. (Nasdaq: SLDB), today provided a first quarter 2021 business update, which includes updates on two additional patients dosed in the IGNITE DMD clinical trial, long term biopsy data from prior patients dosed in the high dose 2E14 vg/kg cohort of the trial, advancement of a next-generation gene therapy program for Duchenne and financial results for the quarter ended March 31, 2021

Biohaven Pharmaceuticals announced that Nurtec® ODT will be the primary partner for the Rick Ware Racing No. 51 NTT IndyCar Series program.

OTO-825 administration rescues hearing and cochlear damage in two preclinical models of congenital hearing loss caused by GJB2 deficiency

Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today reported financial results for the first quarter ended March 31, 2021

Preclinical Data for Congenital Adrenal Hyperplasia and Canavan Disease Programs Shared at American Society of Gene & Cell Therapy Annual Meeting, Enabling Anticipated Clinical Trials This Year

Lyfebulb, a patient-empowerment innovation accelerator that bridges the gap between patient communities and the healthcare industry, and CSL Behring, a global biotherapeutics leader driven by its promise to save and improve lives, are excited to announce that Dr. Minnie Sarwal of NephroSant was selected as the winner of the Lyfebulb-CSL Behring Innovation Challenge: Thriving with Transplantation.