APRIL 2020
Cure Rare Disease has established the necessary infrastructure to enable the development of customized therapeutics for genetic diseases from bench to bedside.
- We have partnered with several industry leading partners to enable preclinical development, manufacturing and therapeutic administration including Charles River Labs, University of Massachusetts Medical School, Viralgen and Aldevron.
- Charles River will lead preclinical development and GLP toxicology studies both of which are critical to reaching patient dosing.
- Viralgen and Aldevron will produce research and clinical grade AAV (viral vector) for systemically delivering the therapeutic to patients.
- The University of Massachusetts Medical School will host the therapeutic infusion once approved by the FDA.
Our first patient is expected to be dosed in late 2020/early 2021 while we have recently enrolled (4) additional DMD patients and (1) LGMD patient into our Cohort 0 to develop customized therapeutics. Additionally, (5) DMD patients were recently enrolled into our Cohort 1 through our Founding Ambassador Program as we seek to scale this endeavor.
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/
- We are engaging the FDA to begin regulatory conversations ultimately leading into clinical trial.
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.
