Cure Rare Disease Welcomes Leading Experts to Scientific Advisory Board

March 20, 2023 |

2 min read

Cure Rare Disease (CRD) - a nonprofit biotechnology company focused on developing treatments for ultra-rare diseases - announces the appointment of leading experts in the fields of gene therapy and neuromuscular disorders to its Scientific Advisory Board (SAB).

WOODBRIDGE, Conn.--(BUSINESS WIRE)-- Cure Rare Disease (CRD) - a nonprofit biotechnology company focused on developing treatments for ultra-rare diseases - announces the appointment of leading experts in the fields of gene therapy and neuromuscular disorders to its Scientific Advisory Board (SAB).

As part of their role, SAB members help advance CRD’s preclinical and clinical development of its gene therapy and antisense oligonucleotide (ASO) programs, providing strategic advice on scientific, regulatory, and clinical matters.

The members of the newly appointed SAB bring a wealth of expertise and experience in the areas of treatment and therapeutic development for neuromuscular diseases. They include:

Nick Johnson, M.D., M.Sci., FAAN (Board Chair)
Adult Neurology, Neuromuscular Vice Chair, Research Associate Professor, VCU School of Medicine
Carsten Bonnemann, M.D.
Senior Investigator, Neuromuscular and Neurogenetic Disorders of Childhood Section, NIH Intramural Research Program
Elizabeth McNally, M.D., Ph.D.
Professor and Director, Center for Genetic Medicine Professor of Medicine
(Cardiology) and Biochemistry and Molecular Genetics, Northwestern Feinberg School of Medicine
Perry Shieh, M.D., PhD
Neurology, Neuromuscular Medicine, UCLA Health
Alan Beggs, Ph.D.
Director, The Manton Center for Orphan Disease Research
Sir Edwin and Lady Manton Professor of Pediatrics, Harvard Medical School

“CRD is honored to welcome these esteemed experts in gene therapy and neuromuscular disorders to our scientific advisory board,” said Rich Horgan, MBA, Founder and CEO of CRD. “Their insights and guidance will be critical as we continue to advance our programs and work towards our goal of providing life-saving treatments to individuals with rare diseases.”

CRD currently has 19 therapeutic programs under development, including potential treatments for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, SCA3 and ADSSL1 distal myopathy, as well as a program aimed at overcoming the challenges of neutralizing antibodies in gene therapy delivery. Collectively, these programs offer pragmatic hope of treatment to more than 200,000 Americans living with rare and ultra-rare diseases.

About Cure Rare Disease
Cure Rare Disease (CRD) is a nonprofit biotechnology company focused on the development of genetic medicines for rare and ultra- rare diseases. CRD is committed to accelerating the development of treatments for individuals with rare diseases through a patient-centric approach to research and development. The company’s programs include gene therapy and antisense oligonucleotide (ASO) treatments for a range of rare diseases. For more information, please visit https://www.cureraredisease.org/.