Weill Medical College Of Cornell University

FDA Grants RMAT Designation to Gene Therapies for Retinitis Pigmentosa

4 months ago

• Bionic Sight's BS01 gene therapy received RMAT designation from the FDA based on Phase 1/2 trial data showing improved light sensitivity and visual acuity in RP patients. • Beacon Therapeutics' laru-zova also received RMAT designation from the FDA for XLRP, supported by Phase 2 DAWN and SKYLINE trials demonstrating improvements in low luminance visual acuity. • RMAT designation expedites the development and review of regenerative medicine therapies, offering benefits like enhanced communication with the FDA and potential for accelerated approval.

Oncolytics Biotech and Bio-Path Holdings Announce Promising Cancer Therapy Advancements

6 months ago

• Oncolytics Biotech presented data at the ASCO GI Symposium, highlighting pelareorep's potential in treating relapsed anal and metastatic pancreatic cancers. • A GOBLET study cohort showed a 33% objective response rate in relapsed anal cancer patients treated with pelareorep and atezolizumab, including a complete response lasting over 15 months. • Bio-Path Holdings reported a meaningful patient response in a Phase 1/1b trial of BP1002 for refractory/relapsed AML, with stable disease and reduced blast count after one treatment cycle. • Bio-Path Holdings' BP1001-A demonstrated continued tumor reduction and stable disease in a solid tumor patient, alongside durable remissions in AML patients treated with prexigebersen.