News

Beam Cleared to Launch Trial of BEAM-101, Gene Editing Therapy

The U.S. Food and Drug Administration (FDA) has cleared Beam Therapeutics to start clinical testing of BEAM-101, the company’s investigational gene editing cell therapy for sickle cell disease (SCD). “BEAM-101 has the potential to offer a one-time treatment for patients with sickle cell disease, and this clearance enables…

‘Best Biotech’ Prix Galien USA Awarded to GBT’s Oxbryta

Oxbryta (voxelotor), Global Blood Therapeutics’ first-in-class oral therapy for sickle cell disease (SCD), has won the 2021 Prix Galien USA Award for best biotechnology product. The award is among the health industry’s most prestigious honors, and recognizes an outstanding product that has improved patient well-being and impacted human…

Emmaus Seeks Approval of Endari in United Arab Emirates

Emmaus Life Sciences is seeking the approval of Endari (L-glutamine), an oral treatment for sickle cell disease (SCD), in the the United Arab Emirates (UAE). “We look forward to working with the U.A.E Ministry of Health to bring this important medication to our patient population in the…

Global Genes, Diversity Coalition Team Up to Advance Health Equity

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…