FDA Awards RMAT Status to AskBio's Parkinson's Gene Therapy Candidate

The US Food and Drug Administration (FDA) has recognized the significance of AskBio's investigational gene therapy for Parkinson’s disease (PD) by granting it Regenerative Medicine Advanced Therapy (RMAT) designation. This designation is particularly reserved for regenerative therapies that are being developed to address serious or life-threatening conditions. The therapy in question, AB-1005, which is a project of Bayer's subsidiary, AskBio, will now enjoy improved regulatory access. This includes the possibility of receiving detailed guidance for efficient drug development, a rolling review process for the Biologics License Application, and other strategies to expedite both development and approval stages.

Parkinson's disease is a progressive neurodegenerative disorder affecting over ten million individuals globally. It is characterized by symptoms such as tremors, muscle rigidity, and slowed movement. One area of interest in PD treatment research has been the use of recombinant glial cell line-derived neurotrophic factor (GDNF). GDNF is a protein known to assist in neuron survival and differentiation, and its potential has been explored in treating conditions marked by the degeneration of midbrain dopaminergic neurons, which are affected in PD.

AB-1005, previously referred to as AAV2-GDNF, has been engineered to provide stable and continuous expression of GDNF in targeted brain regions through direct neurosurgical injection. The FDA's RMAT designation was influenced by evidence from an open-label phase 1b trial of AB-1005. The trial, which extended over 36 months, showed positive trends in clinical outcome measures and confirmed that the therapy was well tolerated, with no serious adverse events related to the product.

The phase 2 study, named REGENERATE-PD, is already underway, with the initial group of patients randomized in the United States. Furthermore, the study is set to expand, with additional sites expected to begin participant enrollment in the US, the UK, Germany, and Poland by the first half of 2025.

Gustavo Pesquin, the chief executive officer of AskBio, expressed enthusiasm about the FDA’s decision, noting the potential benefits for individuals with PD and their families. He emphasized that this milestone could speed up the development of their investigational gene therapy program, underscoring the promising data and potential impact of AB-1005 for patients and the medical field. Pesquin also highlighted the company's eagerness to collaborate closely with the FDA to expedite their program.

Christian Rommel, Bayer's executive vice president and global head of research, echoed this sentiment, pointing out that the RMAT designation for AB-1005 underscores the significant unmet medical needs and the therapy's potential to bring meaningful change to patients with Parkinson’s disease.

In summary, the FDA's RMAT designation for AskBio's AB-1005 represents a pivotal step in the development of an innovative gene therapy aimed at tackling Parkinson's disease. With encouraging trial results and regulatory support, there is hope that this therapy could one day provide significant relief to the millions affected by this challenging condition.