Casma Therapeutics Raises $50 Million to Advance New Therapies for Rare Diseases

Rare Daily Staff

Casma Therapeutics completed a $50 million series B financing to advance its autophagy degrader platform to develop new treatments for patients with rare genetic diseases.

The Column Group led the financings, with participation from new investors Eventide Asset Management, and Schroder Adveq, and undisclosed institutional investors, and existing investor Third Rock Ventures.

“We believe our investors’ strong focus on science, combined with their established position in the life sciences ecosystem, will aid us in continuing to forge ahead as pioneers in this novel area of biology,” said Keith Dionne, CEO of Casma Therapeutics.

Casma Therapeutics is working to accelerate the clinical translation of autophagy insights into new treatments for patients with rare genetic diseases and broader pathologies where restoration of cellular balance may arrest or reverse disease progression. A range of preclinical data supports the idea that boosting autophagy may play an important role in improving cellular clearance in genetic diseases, including lysosomal storage disorders, systemic diseases and CNS disorders.

The company will use proceeds from the financing to advance their TRPML1 agonist program into development for muscular dystrophy, TRPML1 regulates the repair of the plasma membrane of muscle cells following damage, addressing the core pathology in multiple forms of muscular dystrophy.

Casma will also use proceeds from the financing to continue development of its Autophagy Degrader Platform (ADP). ADP is a novel approach for the degradation of multiple disease-causing targets. The autophagy system is able to degrade disease targets substantially larger and more diverse than those that rely on E3 ligase-based or proteasomal degradation approaches.

Photo: Keith Dionne, CEO of Casma Therapeutics