Characterize patient’s genetic mutation and establish a cell line through a tissue biopsy.
Prototype and optimize therapeutic candidates capable of fixing mutation in the cell line.
Test candidates via tissue modeling and/or mutation-specific mouse model for efficacy.
Manufacture final therapeutic & conduct pivotal study. Submit IND to the FDA for approval.
Administer therapeutic and monitor patient(s) for safety and efficacy signals.
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License technology to industry to treat remaining population.
The success of our model relies on our extensive network of academic and industry collaborations across the United States, Canada and Europe.
Our research and development is powered by the generous financial support of families, rare disease communities, and organizations unwilling to accept the status quo of drug development practices. As a 501c3 non-profit biotech, 93+% of all donations go directly to the research and development of life-saving therapies.
CRISPR mediated exon-skipping is a method of genome editing that can be used to develop life-saving therapeutics for people with rare and ultra-rare genetic disorders. CRD is explaining the science behind these technologies as well as how they are the key to treating these diseases.
Today, CRD is breaking down the basics of antisense oligonucleotides (ASOs), which are being utilized to develop life-saving therapeutics for rare neurodegenerative disorders.
Gene therapy is the future of treating rare, genetic diseases that were previously thought to have no cure. CRD is breaking down what gene therapy is and how we are using gene therapy to develop therapies for neuromuscular diseases including Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophy subtypes, and spinocerebellar ataxia type 3 (SCA3).
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