BioSpace News Archive

Preclinical studies conducted with University of Pennsylvania’s Gene Therapy Program demonstrate biologic effects of vector-optimized gene therapy PBKR03 for Krabbe disease PBKR03 showed marked improvements in safety, disease progression and key biomarkers for Krabbe disease in large and small animal models Company is enrolling patients for cohort 1 in global clinical trial, GALax-C, with PBKR03 for infantile Krabbe disease, a rare, fatal pediatric condition PHILADELPHIA, March 29, 2022 (G

Palette Life Sciences, a fully integrated global life sciences company dedicated to improving patient outcomes, announced the results of their Solesta® Post Approval Study.

Renalytix plc (NASDAQ: RNLX) (LSE: RENX) today announced the proprietary Renalytix Accelerating Precision medicine In Diabetes (RAPID) biorepository is on track to exceed 4,000 unique patient biospecimens with matching, deidentified electronic health record data in 2022 through the Company’s real-world evidence program.

Cogent Biosciences, Inc. today announced details for its planned Virtual R&D Investor Event on Friday, April 8, 2022, beginning at 4:05 PM EDT.

Visby Medical™, an innovative diagnostic company, announced its agreement with Urgent Care for Children to offer the company’s new Sexual Health Click Test to patients for the detection of sexually transmitted infections in female patients 14 years of age and older.

European Medicines Agency Validates Bristol Myers Squibb’s Application for Opdivo (nivolumab) with Chemotherapy as Neoadjuvant Treatment for Resectable Non-Small Cell Lung Cancer.

Today, the Cystic Fibrosis Foundation announced an award to Eloxx Pharmaceuticals Inc. for up to $15.9 million in additional funding to support the expansion of its ongoing ELX-02 clinical program focused on developing therapies for people with CF who have nonsense mutations.

Rocket Pharmaceuticals, Inc. today announces that Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, will deliver a virtual company presentation at the Guggenheim 3rd Annual Genomic Medicines and Rare Disease Conference on Friday, April 1 at 8:00 a.m. ET.

Recordati Rare Diseases Announce Publication in T he Journal of Clinical Endocrinology & Metabolism of the Phase III LINC 4 Study Confirming Efficacy and Safety of ISTURISA ® (osilodrostat) in Patients With Cushing’s Disease, The LINC 4 study demonstrated superiority of ISTURISA ® (osilodrostat) over placebo in achieving cortisol control during the 12-week, double-blind, randomized phase (77% vs 8%, P <0.0001). ISTURISA provided rapid and s

Clover Biopharmaceuticals, Ltd., a global clinical-stage biotechnology company developing novel vaccines and biologic therapeutic candidates, reported financial results for the year ended December 31, 2021.