Aurobindo Pharma’s Subsidiary CuraTeQ Secures UK Approval for Bevacizumab Biosimilar

SCG Cell Therapy Partners with A*STAR BTI and NATi to Advance RNA-Based Therapeutics

Korea's National Bio Big Data Project: Advancing Precision Medicine with Macrogen and Illumina

GST Exemption Announced for Cancer Treatment Gene Therapy

AIIMS Develops Affordable CAR-T Cell Therapy for Multiple Myeloma

Sangamo Therapeutics and Astellas Collaborate on Innovative Gene Therapy for Neurological Diseases

FDA Clears IND for Arbor Biotechnologies’ ABO-101 Gene Editing Therapeutic for Primary Hyperoxaluria Type 1

Fortis Healthcare Acquires Stake in Agilus Diagnostics

FDA Approves BRAFTOVI® Combination Regimen as First-Line Treatment for BRAF V600E-Mutant Metastatic Colorectal Cancer

The Role of Big Data in Precision Medicine

Caris Life Sciences Celebrates 1,000+ Precision Medicine Studies, Advancing Cancer Research and Patient Outcomes

OncoHelix and Qualisure Diagnostics Partner to Bring Thyroid GuidePx® Test to Canadian Patients

Gates Foundation Invests $50 Million in Tessera Therapeutics to Advance Gene Therapy for Sickle Cell Disease

FDA Approves Ensartinib (Ensacove) as First-Line Treatment for ALK-Positive NSCLC

Guardant Health and Boehringer Ingelheim Collaborate to Advance Liquid Biopsy CDx for HER2-Targeted Therapy in NSCLC

Home
/
update
/
details

Ascidian Therapeutics Collaborates with Roche to Develop RNA Exon Editing Therapies for Neurological Diseases

NextEdge Admin

19 Jun 2024

3 min 22 sec

Ascidian Therapeutics has entered into a significant research collaboration and licensing agreement with Roche, aimed at advancing RNA exon editing therapeutics for neurological diseases. This partnership grants Roche exclusive rights to Ascidian's RNA exon editing technology for undisclosed neurological targets. Ascidian will lead the discovery and certain preclinical activities in collaboration with Roche, while Roche will take responsibility for additional preclinical work, clinical development, manufacturing, and commercialization.

Under the terms, Ascidian will receive an initial payment of $42 million and could potentially earn up to $1.8 billion in milestone payments related to research, clinical achievements, and commercial sales, along with royalties on global sales. Importantly, Ascidian retains the freedom to pursue programs against other neurological targets independently or with different partners.

Michael Ehlers, M.D., Ph.D., President and CEO of Ascidian Therapeutics, expressed pride in the scientific rigor that led to this partnership, highlighting the potential of large-scale RNA exon editing to address diseases currently underserved by gene editing technologies. He emphasized Ascidian's commitment to relieving suffering and enhancing lives through innovative therapeutic approaches.

Roche's Global Head of Pharma Partnering, James Sabry, M.D., Ph.D., acknowledged the opportunity to leverage Ascidian's advanced RNA exon editing platform. This technology holds promise for delivering transformative therapies by editing multiple exons at the RNA level in a single treatment, potentially offering durable therapeutic benefits with reduced risks compared to DNA editing.

Ascidian's RNA exon editing platform is designed to target large and highly mutable genes while preserving natural gene expression patterns. By focusing on RNA rather than DNA, Ascidian aims to combine the lasting effects of gene therapy with enhanced safety profiles.

Currently, Ascidian is actively progressing its clinical pipeline, including ACDN-01, the first RNA exon editing candidate targeting Stargardt disease and related conditions. This therapy recently received U.S. FDA Investigational New Drug (IND) clearance, with ongoing Phase 1/2 clinical trials underway.

Through this collaboration, Ascidian and Roche aspire to pioneer novel treatments that offer patients and their families new hope in managing complex neurological diseases with potentially groundbreaking RNA exon editing medicines.