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...
Latest News
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...
AUSTIN, Texas — Rein Therapeutics (“Rein”) (NASDAQ: RNTX), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, today announced the publication of novel data on its lead drug candidate, LTI-03, in iScience, a peer-reviewed, open-access journal published...
AUSTIN, Texas — Rein Therapeutics (“Rein”) (NASDAQ: RNTX), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, today announced that it has received authorization from the European Medicines Agency (EMA) to initiate the Company’s Phase 2 “RENEW”...
AUSTIN, Texas — Rein Therapeutics (“Rein”) (NASDAQ: RNTX), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, today announced that the U.S. Food and Drug Administration (FDA) has lifted the full clinical hold on the Company’s Phase...
AUSTIN, Texas — Rein Therapeutics (“Rein”) (NASDAQ: RNTX), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, announced that a new preprint has been posted to medRxiv describing clinical and translational findings from an inhaled, dose-escalation study of...
AUSTIN, Texas — Rein Therapeutics (“Rein”) (NASDAQ: RNTX), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has authorized the Company to initiate its Phase...