FALL 2020
Our research took a BIG step towards the clinic! We’ve provided a summary of the accomplishments below.
FDA Pre-IND Meeting
- Successful PreIND meeting with FDA on September 29th
- Positive comments received regarding study plan
- PreIND guidance provides template for all future customized therapeutics
Neutralizing Antibodies (NAb) Initiative established
- NAbs prevent the successful delivery of AAV drugs including CRISPR and micro-dystrophin. Currently estimated at 30-50% of the population and a major hurdle.
- NAb analysis program established to measure patient NAb levels. Five samples analyzed thus far with a target of 200.
- Formed NAb technical steering committee to help guide development of technologies to suppress NAbs in 1-2 years
Additional Updates
- GMP therapeutic under production – expected: December 2020
- Preclinical road map established for 3 additional patients
- Targeted dosing of patient 1 is first half of 2021
We need the community’s help to increase our capacity to treat more patients with customized therapeutics.
Find out how you can help support us: https://cureraredisease.org/help-fund-treatments/
The purpose of Cure Rare Disease is to end rare disease. There are over 7,000 different rare diseases impacting over 300 million people worldwide.
Drug development for rare disease is challenging. The “one size fits all” approach to drug development is not sufficient.
In 2018, the world saw one of the first examples of the power of personalized medicine to save those with complex genetic diseases for which there are no treatments.
Cure Rare Disease has formed a collaboration with the leading researchers to develop a customized therapeutics platform, in time to save our loved ones.
The approach that Cure Rare Disease is pursuing is unprecedented. By aligning researchers around a clear goal with foundation support and oversight promotes accountability, takes advantage of group wisdom to enable rapid development without traditional drawbacks.
Collaboration among our researchers enables faster innovation.
Our collaboration brings together researchers and clinicians who are luminaries and pioneers.
Monkol Lek, PhD
Yale Medical School
Louis Kunkel, PhD
Boston Children’s Hospital
Stanley Nelson, MD
David Geffen School of Medicine at UCLA
Charles Emerson, PhD
University of Massachusetts Medical School
Brenda Wong, MD
University of Massachusetts Medical School
Keryn Woodman, PhD
Yale Medical School
Angela Lek, PhD
Yale Medical School
Moreover, Cure Rare Disease has the support of leaders in the space who are pioneers in developing customized treatments.
ADVISORY TEAM
Timothy Yu, PhD, MD
Boston Children’s Hospital
Jamie Heywood
ALS Therapy Development Institute & Patients Like Me
Jonathan Fleming
QurAlis
THE STRATEGY TO END DUCHENNE
Our strategy is to create a common foundation from which our collaborators can take multiple therapeutic “shots on goal”.
The process is 4-fold and begins with establishing a cell line unique to the individual. With this, we can better analyze the genetic mutations and, downstream, test therapeutic candidates for efficacy against the individual’s cells.
The analytical phase guides the subsequent stages of therapeutic development. Our team will use computational prediction to guide which of the therapeutic “tools” will be most effective at restoring functional protein levels based on the individual’s genetics.
Of the therapeutic candidates, the lead candidate will be developed and optimized. Once complete, we will move into a single (or small scale) clinical trial.
Our goal is to be in clinical trial in 1-2 years.
In parallel, we will begin to generalize the process by creating cohorts of different mutation groups and include other individuals.
The vision of Cure Rare Disease is to show that this model can work and then drive towards pay or approval so that all families can benefit.
Have more questions about our drug development process?
Watch our founder, Rich Horgan, explain the Cure Rare Disease customized therapeutics approach in a one-hour long webinar.
