Key Highlights
- Charles River Laboratories and Genetic Cures for Kids Inc (GC4K) announce a vital collaboration to develop a gene therapy for Hereditary Spastic Paraplegia Type 56 (SPG56).
- GC4K, founded by the parents of a child affected by SPG56, aims to not only cure SPG56 but also create a model for treating other genetic diseases.
- Charles River’s eXpDNA™ platform and plasmid DNA expertise will be pivotal in advancing the development of this potential cure.
Source: Business Wire
Notable Quotes
- “The opportunity to work with GC4K is exactly why we do what we do, playing a role in delivering potentially curative treatments to patients suffering with an ultra-rare disease. We are excited to support the team as they work through the next stage of their program development.” – Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions at Charles River
- “What was once a tragic diagnosis, has the potential to become a triumph, as we draw nearer to finding a cure for SPG56. Our partnership with Charles River is bringing us closer to aiding children like Tallulah, who, until now, have faced the daily hardships of SPG56 without any glimmer of hope.” – Golden Whitrod, Tallulah Moon’s mother and Co-Founder and President at Genetic Cures for Kids, Inc
SoH's Take
This collaboration between Charles River Laboratories and Genetic Cures for Kids signifies a significant leap forward in the realm of rare genetic disease research. The use of Charles River’s advanced plasmid DNA technology, coupled with the passionate drive of GC4K, could potentially lead to a breakthrough in treating SPG56 – a condition that has long eluded effective treatment options. Furthermore, this partnership holds the promise of establishing a model that can be replicated to tackle other rare genetic conditions, addressing a critical gap in current medical research and offering hope to thousands of families worldwide. The commitment of both organizations to this cause underscores the increasing importance of collaborative efforts in the field of gene therapy and rare disease research, marking a potentially transformative moment in medical history.
