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Customized Therapeutics

August 2019 | Research & Development Update

Cure Rare Disease has finished developing the infrastructure necessary to enable continuous development of precision medicine therapeutics for genetic diseases

  • We have onboarded two critical partners to enable continuous development: Charles River Labs and Nationwide Children’s Hospital.
  • Charles River will lead regulatory engagement and GLP toxicology studies both of which are critical to reaching patient dosing.
  • Nationwide Children’s Hospital will produce research and clinical grade AAV (viral vector) for systemically delivering the therapeutic to patients.

Our first patient is expected to be dosed in summer 2020 while we have recently enrolled (2) additional DMD patients into the precision medicine process.

  • We have engaged 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.

Researchers & Clinicians

Timothy Yu, PhD, MD

Boston Children’s Hospital 

Louis Kunkel, PhD

Boston Children’s Hospital 

Monkol Lek, PhD

Yale Medical School

Stanley Nelson, MD

David Geffen School of Medicine at UCLA 

Angela Lek, PhD

Yale Medical School

Charles Emerson, PhD

University of Massachusetts Medical School

Brenda Wong, MD

University of Massachusetts Medical School

Keryn Woodman, 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

Kevin M. Flanigan, MD

Nationwide 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.
We encourage interested individuals and groups to reach out. Time is of the essence. If you wish to support this groundbreaking initiative:
DONATE HERE
​Contact: info@cureRD.org
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