SAN DIEGO — Locanabio, Inc., a genetic medicines company developing RNA-targeted therapeutics for patients with rare genetic neuromuscular and neurodegenerative diseases, announced that CureDuchenne Ventures, the strategic investment arm of CureDuchenne, has made an equity investment in Locanabio. This investment will support the development of the company’s snRNA-mediated exon skipping...
treatment News
LEXINGTON, Mass. – LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced an update on screening and enrollment activities for the SUNRISE Phase I/II clinical trial of LB-001, the company’s investigational treatment for...
LEXINGTON, Mass. – LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today announced that the first patient has been dosed with LB-001, the Company’s investigational single-administration gene editing therapy based on its proprietary...
LEXINGTON – LogicBio Therapeutics, Inc. (Nasdaq: LOGC), a clinical-stage genetic medicine company, today provided an update on the LB-001 clinical development program. The U.S. Food and Drug Administration (FDA) has notified the company that its Phase 1/2 SUNRISE clinical trial of LB-001 in pediatric patients with methylmalonic acidemia (MMA) has...
WHIPPANY, N.J.- Bayer to present new data across four distinct analyses showcasing the consistent, long-term clinical profile for Vitrakvi® (larotrectinib) across TRK fusion cancer patients of all ages (range: 0.1-84 years) and multiple tumor types. The analyses include updated long-term efficacy and safety data across solid tumors, including primary central...
Nashville, Tennessee – A 10-year follow up study of nearly 2,500 U.S. men who received prostate cancer treatment will help inform decision making in terms of treatments and side effects for a diverse population. The CEASAR (Comparative Effectiveness Analysis of Surgery and Radiation for Localized Prostate Cancer) study, coordinated by...
Cleveland, Ohio – Older patients with Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) who were not good candidates for the standard treatment of intensive chemotherapy had a median overall survival (OS) of 6.5 years on an alternate regimen of dasatinib and blinatumomab. These long-term results from the S1318 clinical trial...
MIAMI — Longeveron Inc. (NASDAQ: LGVN NASDAQ: LGVNR) (“Longeveron” or “Company”), a clinical-stage biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions such as hypoplastic left heart syndrome (HLHS), Alzheimer’s disease and Aging-related Frailty, announced today that extended long-term follow-up data from the Company’s ELPIS I trial of...
The recombinant factor VIII (FVIII) therapy BAY 94-9027 demonstrated long-term safety and efficacy at reducing bleeds in pediatric patients with severe hemophilia A, according to results of a long-term analysis published in the journal Haemophilia. The analysis was the extension study of the phase 3 PROTECT VIII Kids trial (ClinicalTrials.gov Identifier: NCT01775618),...
LA JOLLA, Calif. — Longboard Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced that it will host a conference call to discuss topline data from the PACIFIC Study, a Phase 1b/2a clinical trial evaluating bexicaserin (LP352) in participants with a broad...