Raj Mehra, PhD, and Krishna Subramanian, PhD, on Striving For Disease-Modifying Therapies in Parkinson Disease

“The biggest unmet need for Parkinson's, currently, is there are no disease modifying therapies [DMTs] available; almost all the approved drugs are mostly symptom relief. And now that we have started to understand, at the genomic levels, different mutations that lead to Parkinsonism, we should be focused on the DMTs.”

While there are treatments available for Parkinson disease (PD), these treatments generally only ameliorate symptoms, and are not disease modifying. As research brings the genetic factors underlying PD into clearer focus, gene therapy approaches that treat the root cause of the disease are becoming an area of increased interest.

In an interview with CGTLive, Raj Mehra, chairman and chief executive officer, Seelos Therapeutics, and Krishna Subramanian, PhD, head of non-clinical and translational science, Seelos Therapeutics, discussed SLS-004, the company’s preclinical gene therapy candidate for the treatment of PD. Mehra spoke about underlying factors in PD disease pathology, and why the gene that codes for α-synuclein was selected as the therapeutic target for SLS-004. He also detailed the encouraging results seen so far in mouse model research with SLS-004.

Image depicting modulation of SNCA with AAV-A53T + SLS-004 vector.

Subramanian expanded on the specifics of the mouse model used in the research. He emphasized the importance of using a humanized mouse model and humanized vector model in order to obtain data more likely to translate to future clinical research. He also spoke about SLS-004's epigenetic approach to modulating gene expression, which could help avoid some of the safety issues associated with direct changes to genetic material.