Cure Rare Disease Receives FDA Feedback on Limb-Girdle 2i/R9 Gene Therapy Program Following Successful Pre-IND Meeting
Cure Rare Disease (CRD), a nonprofit biotechnology organization developing genetic therapies for ultra-rare diseases, today announced the successful completion of a pre-Investigational New Drug (pre-IND) meeting with the U.S. Food and Drug Administration (FDA) regarding its investigational gene therapy program for Limb-Girdle Muscular Dystrophy type 2I/R9 (LGMDR9).
Cure Rare Disease Welcomes New Scientific and Strategic Leaders to Advance Therapies for Ultra-Rare Conditions
Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology organization developing genetic therapies for ultra-rare conditions, is proud to announce the appointment of Keith Sutton, PhD, as Chief Science Officer and the addition of Michelle Werner to its Board of Directors. These strategic additions bring deep scientific expertise and seasoned biopharmaceutical leadership to CRD as the organization continues to advance therapeutic development for rare disease patients.
Cure Rare Disease Receives Orphan Drug Designation from FDA for Investigational Therapy Targeting Spinocerebellar Ataxia Type 3 (SCA3)
Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company developing genetic medicines for ultra-rare and rare conditions, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational anti-sense Oligonucleotide therapeutic for the treatment of Spinocerebellar Ataxia (SCA), including Spinocerebellar Ataxia Type 3 (SCA3), a progressive and currently untreatable neurodegenerative disorder.
We Row For William: 157 Miles, One Nonstop Journey of Hope
On April 25, Joe Jackson and a dedicated crew of men including Jeremy Leever and Shaun Olson will embark on an extraordinary nonstop, overnight rowing journey covering 157 miles of the Rogue River to raise awareness and critical funds for his son, William Jackson, who is battling Duchenne muscular dystrophy (DMD).
Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3), a neurodegenerative disorder with no current treatment.
Cure Rare Disease Appoints Brittany Stineman as Chief Advancement Officer
Cure Rare Disease, a nonprofit biotechnology organization dedicated to advancing treatments for neglected rare and genetic diseases, is proud to announce Brittany Stineman as its new Chief Advancement Officer, effective January 2025.
Cure Rare Disease Expands Leadership Team and Board of Directors to Advance Mission
Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology organization, announces the addition of a Chief Advancement Officer to its leadership team and welcomes three new members to its board of directors. Markus Ward joins as Chief Advancement Officer, and Jeremy Sternberg, J.D., Klaus Veitinger, M.D., Ph.D., and Mike Williams join the Board of Directors.
Cure Rare Disease Welcomes Leading Experts to Scientific Advisory Board
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).
Cure Rare Disease Receives Muscular Dystrophy Association Grant
Cure Rare Disease, a clinical-stage nonprofit biotechnology company, has been awarded an advocacy collaboration grant from the Muscular Dystrophy Association (MDA) to support research on novel reimbursement strategies for drugs developed for ultra-rare diseases, a key challenge for rare disease patients, including muscular dystrophy patients. As reimbursement decisions are dependent upon commercial approval through large clinical trials, potential therapies developed for the rare and ultra-rare diseases are unlikely to reach the threshold of criteria to support reimbursement.
Cure Rare Disease Acquires Laboratory Space for New Headquarters in Connecticut
Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company, has acquired its first laboratory space at 4 Research Drive in Woodbridge, Connecticut, where the organization will relocate its headquarters and expand its research and development operations.
Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic
Cure Rare Disease (CRD) - a Boston-based 501c3 nonprofit biotech - announces the approval from the U.S. Food and Drug Administration (FDA) to administer its very first therapeutic. The drug, named CRD-TMH-001, treats muscle promoter and exon 1 mutations on the dystrophin gene. With the IND approval process complete, the FDA has given the go-ahead, and dosing of the drug will occur imminently at UMass Chan Medical School.
