Myrtelle Inc., a clinical-stage gene therapy company focused on neurodegenerative diseases, announced its upcoming participation in a panel discussion at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting. The session, titled "FDA's START Pilot Program in Action: Insights from Year One," will take place on Thursday, May 15, 2025, at 3:45 PM CDT in New Orleans.
The panel will highlight Myrtelle's experience as one of just three gene therapy developers selected by the FDA's Center for Biologics Evaluation and Research (CBER) for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.
Accelerating Rare Disease Therapy Development
The START Pilot Program represents a significant shift in regulatory engagement for rare disease treatments. It facilitates more frequent and flexible communications between therapy developers and the FDA, moving beyond traditional meeting structures to accelerate development timelines for urgently needed treatments.
Adrian Stecyk, CEO at Myrtelle, emphasized the program's impact: "The FDA's commitment to accelerating treatments for devastating rare diseases is not just inspiring—it's lifesaving. By working side-by-side with companies like ours, they are helping to bring hope and momentum to communities that have waited far too long."
Advancing Treatment for Canavan Disease
Myrtelle's participation in START has already shown tangible benefits for the development of its first-in-human gene therapy for Canavan disease (CD)—rAAV-Olig001-ASPA. Through the program, the company receives timely regulatory guidance and support, enabling more efficient navigation of the clinical development process.
Canavan disease is a fatal childhood genetic brain disorder caused by mutations in the ASPA gene, which prevent normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. The condition negatively impacts brain development and function, with symptoms typically appearing several months after birth.
Children with Canavan disease experience poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones. As the disease progresses, patients develop seizures, spasticity, swallowing difficulties, and overall muscle deterioration, with most affected children facing life-threatening complications by approximately 10 years of age. Currently, only palliative treatments are available.
Innovative Gene Therapy Approach
Myrtelle's gene therapy is designed to address the root cause of Canavan disease by delivering a functional ASPA gene using a proprietary oligodendrocyte-targeting AAV vector. This approach aims to restore normal aspartoacylase expression, potentially improving brain development and quality of life for affected children.
The therapy's potential has been recognized through multiple regulatory designations, including:
- RMAT, Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA
- Orphan Drug Designation and ATMP classification from the European Medicines Agency
- Innovative Licensing and Access Pathway (ILAP) status from the UK Medicines & Healthcare products Regulatory Agency
Global Recognition and Strategic Partnerships
Myrtelle's innovative approach to treating neurodegenerative diseases has garnered international attention. The company has established an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program, further validating the potential of its therapeutic approach.
The company's proprietary platform, intellectual property, and portfolio of programs support novel gene therapy approaches for neurodegenerative diseases beyond Canavan disease, positioning Myrtelle as a leader in this therapeutic area.
Session Details and Clinical Trial Information
The ASGCT panel session will be held at the New Orleans Ernest N. Morial Convention Center in Room 278-282 from 3:45 pm to 5:30 pm on May 15th, 2025.
For those interested in learning more about Myrtelle's clinical trial for Canavan disease, information can be found on ClinicalTrials.gov under the identifier NCT04833907.
