RARECast

Susan Ruediger, founder and CEO of CMT Research Foundation, discusses her organization’s efforts to catalyze drug development for Charcot-Marie Tooth disease, what it’s done to address obstacles, and some of the partnerships it has established to advance the development of treatments and potentially a cure.

Murat Kalayoglu, president and CEO of Cartesian Therapeutics, discusses the company’s RNA-engineered cell therapies, how they work, and how the company is pushing the use of cell therapies beyond cancer.

Richard King, CEO of Spruce Biosciences, discusses the rare endocrine disorder CAH, how his company’s experimental therapy tildacerfont works, and what the company is doing to build a pipeline of other rare endocrine therapies behind it.

Tim Miller, co-founder and CEO of Forge Biologics, discusses the company’s hybrid business model, how it hopes to differentiate itself through its shared experience with its customers, and its emerging pipeline of gene therapies to treat rare diseases.

Daniel Gruskin, chief medical officer of LogicBio, discusses the company’s platform technology, the advantages it provides, and the company’s lead experimental therapy for the rare metabolic condition methylmalonic acidemia.

Nasha Fitter, vice president of rare disease for Ciitizen, about her own experience as a mother of a child with a rare neurologic condition, her work as a rare disease advocate, and a collaboration between a group of rare neurologic disease advocacy organizations to build a natural history study on the Ciitizen platform.

Bruce Goldsmith, CEO of Passage Bio, discusses the company’s relationship with Penn’s Gene Therapy Program, its focus on CNS conditions, and the company’s lead program in the rare lysosomal storage disorder GM1 gangliosidosis.

Cedric Francois, co-founder and CEO of Apellis, discusses the recent FDA approval of the PNH therapy Empaveli, what advantages it may provide over existing therapies, and other indications the company will pursue for this medicine.

Eric Richman, CEO of Gain Therapeutics, discusses its platform technology, how it works, and why it offers a compelling alternative to other approaches to treating lysosomal storage disorders.

Cat Lutz, senior director of mouse repository and in vivo pharmacology genetic resource science at The Jackson Laboratory, discusses the role mouse models play in rare disease research, how new gene editing technologies are changing the development of mouse models, and why new technologies are unlikely to displace the critical role they play anytime soon.