A Positive Outlook for Rare Disease Research: Michael Singer, MD, PhD


The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.

“Anytime I talk to any patient, or friend or family member, who's received a difficult diagnosis, I think one of the most important things you can do for them is let them know that, in addition to everything that doctors are doing, there's a lot of scientists and clinical researchers working to come up with new solutions for them. And sometimes that hope really contributes to the patient's outcomes.”

Monday, February 28, marked international Rare Disease Day, recognizing over 7000 rare conditions that affect over 300 million people worldwide. The day helps to bring researchers, patients, advocates, and policy makers together in conversations on how to improve awareness and management of rare diseases.

One such rare disease is myasthenia gravis, which Cartesian Therapeutics is currently targeting with their lead developmental candidate, the autologous RNA T-cell therapy Descartes-08. The cell therapy, which is directed against B-cell Maturation Antigen is being evaluated in a phase 1/2 study (NCT04146051). Interim data from the study previously showed improvements on the Myasthenia Gravis Foundation of America Clinical Classification and Myasthenia Gravis Composite scales.

CGTLive spoke with Michael Singer, MD, PhD, cofounder chief scientific officer, Cartesian, and to learn more about the importance of Rare Disease Day and why collaboration and advocacy is so important to help improve awareness in this space.

Cartesian Therapeutics announces clinical responses in first cohort of phase 1b/2a trial in myasthenia gravis. News release. Cartesian Therapeutics. August 24, 2021. https://www.cartesiantherapeutics.com/cartesian-therapeutics-announces-clinical-responses-in-first-cohort-of-phase-1b-2a-trial-in-myasthenia-gravis/
Related Videos
Brian Van Tine, MD, PhD, on Looking Ahead on Cell Therapy for Sarcomas
Dilsher Dhoot, MD, on a Promising Start With Diabetic Retinopathy Gene Therapy RGX-314
J. Andrew Livingston, MD, on Forging Forward With Novel Sarcoma Trials
Shankar Musunuri, PhD, on Gene Modifier Therapy vs Gene Replacement Therapy for IRDs
Pat Furlong, BSN, RN, on the State of Gene Therapy in Muscular Dystrophy
Michael Kelly, PhD, on Continuing Progress With Gene Therapy in Muscular Dystrophy
© 2023 MJH Life Sciences

All rights reserved.