AskBio Receives FDA Regenerative Medicine Advanced Therapy Designation for Parkinson’s Disease Gene Therapy

AskBio, a gene therapy company wholly owned by Bayer AG, has announced that its investigational gene therapy AB-1005 for Parkinson’s disease has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the United States Food and Drug Administration (FDA). This designation follows 36-month data from a Phase Ib study demonstrating a favorable safety profile and positive clinical trends. Promising Data and Clinical Development AB-1005, formerly known as AAV2-GDNF, is an investigational gene therapy designed to slow disease progression and improve motor outcomes in patients with moderate-stage PD. The FDA’s decision to grant RMAT designation was based on data from AskBio’s open-label, uncontrolled Phase Ib trial, which showed that AB-1005 was well tolerated, with no serious adverse events related to the therapy. Additionally, clinical outcomes demonstrated trends in improvement or stability across key Parkinson’s disease assessment scales, including the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and patient-reported motor diaries. Some participants also exhibited reductions in Parkinson’s medication usage. Accelerating Innovation Through RMAT Designation RMAT is a special designation granted by the FDA to regenerative therapies, including gene therapies, that demonstrate the potential to address unmet medical needs for serious or life-threatening diseases. This designation offers benefits such as increased FDA guidance, priority review processes, and the possibility of an expedited regulatory pathway. “The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with Parkinson’s disease and their loved ones,” said Gustavo Pesquin, CEO of AskBio. “This milestone could potentially expedite the development of our investigational gene therapy program, and it highlights the promise of AB-1005 for both patients and the broader medical community.” Christian Rommel, Executive Vice President and Global Head of Research and Development at Bayer, added, “The RMAT designation underscores the high unmet medical need in Parkinson’s disease and reinforces our commitment to delivering breakthrough innovations.” Ongoing Clinical Trials and Next Steps Enrollment is ongoing for the Phase II REGENERATE-PD clinical trial, with the first participants already randomized in the United States. Additional study sites in Germany, Poland, and the United Kingdom are expected to open in the first half of 2025. AB-1005 remains an investigational therapy and has not been approved by any regulatory authority. Its safety and efficacy continue to be evaluated in ongoing clinical trials. AskBio remains committed to advancing its research to provide potential new treatment options for Parkinson’s disease patients worldwide. Photo: AskBio