Since its early days in the mid-2000s, optogenetics, with its potential to activate neurons with light, emerged as a promising technique for restoring vision in blind patients. In recent years, at least two companies have announced the start of clinical trials to test optogenetics-based therapies in humans, and one of...
treatment News
Carlene Knight would love to do things that most people take for granted, such as read books, drive a car, ride a bike, gaze at animals in a zoo and watch movies. She also longs to see expressions on people’s faces. “To be able to see my granddaughter especially —...
SAN DIEGO, Calif. — BlossomHill Therapeutics, Inc., a privately-held, clinical-stage biopharmaceutical company applying an intentional, chemistry-based approach to develop innovative small molecule medicines for the treatment of cancer, today announced that initial dose escalation data from its Phase 1/1b trial of BH-30236, a macrocyclic CDC-link kinase (CLK) inhibitor, in relapsed...
North Chicago, Illinois — BLR Bio, an emerging biotechnology company in Rosalind Franklin University’s Helix 51 biomedical incubator, announced that the U.S. Food and Drug Administration (FDA) granted an Orphan Drug Designation for its investigational therapy BLR-200 for the treatment of systemic sclerosis (SSc), also known as scleroderma. Orphan Drug...
NORTH CHICAGO, Ill. — BLR Bio, an early stage biotech company in Rosalind Franklin University’s Helix 51 biomedical incubator, released new data on its novel cancer therapy recently at the 7th annual Labroots Drug Discovery and Development conference. The data points to a promising strategy to boost the effectiveness of...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel) for priority review. Beti-cel is a potentially transformative gene therapy for adult, adolescent and pediatric patients with β-thalassemia across all genotypes who require regular red...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) today announced that the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded based on the review of all available data that the benefit-risk balance of medicinal products containing ZYNTEGLO™ (betibeglogene autotemcel gene therapy) remains favorable. As of today, bluebird bio has...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin for sickle cell disease (SCD) gene therapy (bb1111) for adult and pediatric patients with SCD,...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) announced new data from the clinical development program for its investigational elivaldogene autotemcel (eli-cel, Lenti-D™) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including updated results from the pivotal Phase 2/3 Starbeam study (ALD-102) and the long-term follow-up study LTF-304, as well...
CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE) announced today business and program updates across its severe genetic disease portfolio including a revised diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its Phase 1/2 study of LentiGlobin for sickle cell disease (SCD) (bb1111), the company’s decision to withdraw...