CAMBRIDGE, Mass. – October 6, 2022 – Caraway Therapeutics today announced that the Company will be presenting new data at The Michael J. Fox Foundation’s Parkinson’s Disease Therapeutics Conference, taking place October 13, 2022, in New York, New York. Magdalene Moran, Ph.D., President & Chief Scientific Officer of Caraway and Donato del Camino, Ph. D., Vice President of Drug Discovery will present the poster “Advancing TRPML1 Agonists for GBA-PD”, detailing the Company’s TRPML1 program currently in preclinical development for GBA-Parkinson’s disease (GBA-PD).
“We are excited to provide updates on our ongoing efforts to develop TRPML1 activators as potential therapeutics for GBA-Parkinson’s disease. Our poster presentation highlights data indicating that TRPML1 activation enhances the activity of the glucocerebrosidase (GCase) pathway and alters the clearance of both disease-relevant lipids and alpha-synuclein, key drivers of GBA-PD disease processes,” said Dr. Moran. “As we continue to advance our TRPML1 GBA-PD program, we are committed to bringing new therapeutic options to patients living with GBA-Parkinson’s disease. We remain grateful to The Michael J. Fox Foundation for their continued support.”
GBA-PD is the most common genetic form of Parkinson’s disease. Patients with GBA mutations tend to have earlier onset of disease, more rapid progression, and a higher risk of dementia than patients with idiopathic PD. Homozygous mutations in glucocerebrosidase, the protein encoded by GBA, cause the lysosomal storage disease, Gaucher disease, indicating that GBA is critical to healthy lysosomal function.
TRPML1 is a non-selective cation channel that contributes to the regulation of lysosomal ionic contents and mediates communication with the rest of the cell. Data suggest that enhancing TRPML1 activity improves lysosomal function, and in previous research Caraway’s TRPML1 activators have enhanced GCase activity and expression in both models of lysosomal storage diseases as well as in autosomal dominant polycystic kidney disease (ADPKD).
Poster presentations will be given throughout the day of the conference. More information on the event can be found at https://www.michaeljfox.org/2022-pd-therapeutics-conference.
About Caraway Therapeutics
Caraway Therapeutics is a biopharmaceutical company pursuing novel approaches for the treatment of genetically defined neurodegenerative and rare diseases. The Company is a leader in the cutting-edge science of activating cellular recycling processes to clear toxic materials and defective cellular components by modulating lysosomal function. Caraway is utilizing its unique product engine to develop proprietary insights into lysosomal function and small molecule ion channel modulation and advance a robust pipeline of precision therapeutic candidates with disease-modifying potential for patients. The Company is backed by top-tier investors, including SV Health Investors, AbbVie Ventures, MRLV Fund, Amgen Ventures, Dementia Discovery Fund, Alexandria Venture Investments, and Eisai Innovation.
Caraway is based in Cambridge, MA. For more information, please visit www.carawaytx.com.
Kari Watson or Carolyn Noyes