INDIANAPOLIS — A study of more than 21,000 average risk patients at 186 sites across the U.S., led by Regenstrief Institute and Indiana University School of Medicine research scientist Thomas Imperiale, M.D., has found that the next generation multi-target stool DNA colorectal cancer screening test detects 94 percent of colorectal...
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ROCKVILLE, Md., – Potential one-time gene therapy for the treatment of Duchenne includes a novel, optimized microdystrophin transgene and REGENXBIO’s proprietary NAV® AAV8 vector Innovative trial design, including comprehensive immunosuppressive regimen, to evaluate safety and optimal dose cGMP process material made at commercial-scale to be used throughout clinical development of RGX-202...
ROCKVILLE, Md. –– REGENXBIO Inc. (Nasdaq: RGNX) today announced the publication of results from the Phase I/IIa trial evaluating the safety and tolerability of a single dose of subretinal ABBV-RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). Two-year data were published in The Lancet in a paper titled...
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...
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...