Our Process
Explore our five step process when taking on a new disease.
Select disease, identify collaborator, establish sponsored research agreement
Prototype and develop therapeutic and
in-vitro/in-vivo proof of concept
Test candidates and move into translational
development/CROs
Submit IND to FDA and begin clinical trials
License treatment or seek PRV to open up potential revenue stream
Selection Process
CRD looks at a series of four criteria when selecting conditions to consider for treatment: Indication, Modality, Research Availability, and Patient Population Size.
If all four conditions are present,
CRD could move forward with treatment plans.
CRD looks at a series of four criteria when selecting conditions to consider for treatment: Indication, Modality, Research Availability, and Patient Population Size.
Is the disease a neuromuscular or neurodegenerative condition?
Is the condition treatable with ASO, gene replacement or gene editing?
Are the appropriate researchers and research tools (cell models, animal models, etc) available?
Does the condition have a patient population size of over 50 patients?
If all four conditions are present,
CRD could move forward with treatment plans.
Our Platform Technologies
Our Platform Technologies
CRISPR, or “Clustered Regularly Interspaced Short Palindromic Repeats,” allows scientists to modify DNA. Cure Rare Disease designs and develops gene editing therapies using CRISPR technologies to treat specific genetic mutations.
AAV is a promising new vector for gene therapy, delivering genetic material directly into cells. Recent advances have led to significant improvements in targeting precision and efficiency, allowing for more personalized treatments.
Today, CRD is breaking down the basics of antisense oligonucleotides (ASOs), which are being utilized to develop life-saving therapeutics for rare neurodegenerative disorders.
Interested in collaborating with Cure Rare Disease? Complete the form to connect with a member of our team.
Cure Rare Disease, a non-profit organization (Tax ID number 82-2473513), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.