Work With Us

Join Our Mission to Develop Life-Saving Therapies for Rare Neuromuscular Diseases

Our Drug Development Model

The success of our model relies on our extensive network of academic and industry collaborations across the United States, Canada, and Europe. As our scope of our research and development grows, so does our need to expand our network of collaborators to advance therapeutics for rare and ultra-rare neuromuscular diseases, speed drug development for orphan diseases, and expand access to treatments to broader populations.


For translational researchers and clinicians in the rare neuromuscular space who are interested in advancing therapeutics into the clinc, we invite you to connect with Cure Rare Disease to explore these opportunities for rare and ultra-rare neuromuscular indications.


We offer a number of opportunities for collaboration with de novo and later-stage development programs, including the ability to explore a technology in the rare neuromuscular space and the opportunity to license technologies which have been de-risked in limited patient populations.


We leverage our development infrastructure to partner with foundations who understand the needs of their patient population and who want to develop a therapeutic for the indication or subset of the indication.

Jim Foster

CEO, Charles River

"Charles River is proud to partner with Cure Rare Disease, an institution that is accelerating a unique drug development model that supports even the rarest of patients. The depth of technical sophistication, collaboration and transparency that CRD embodies is critical to advancing high-impact modalities for patients who urgently need them, and ultimately has the potential to benefit society-at-large."

Monkol Lek, PhD

Assistant Professor of Genetics, Yale Medical School

"Working with Cure Rare Disease to prototype and optimize therapeutics drastically helps to accelerate the drug development process for ultra-rare disease. Moreover, CRD's collaborative framework of regulatory consultants and CRO partners effectively closes the gap between academic science and the ultimate goal of getting to the patient bedside. This unique and unparalleled support is something that does not exist broadly within the field."

Keith Sutton, PhD

Scientific Advisor, Charles River

"Cure Rare Disease's focus on ultra-rare, collaboration-based drug development harnesses the power of large partners like Charles River with innovations from academics, making it possible to accelerate ultra-rare therapeutic needs. Further, as CRD and their partners leverage learnings from small-scale trials, they will undoubtedly benefit larger disease populations – impacting thousands of patients now and in the future."

Alan Beggs, PhD

Director of the Manton Center for Orphan Disease Research, Boston Children’s Hospital

"When a family impacted by an ultra-rare disease approached my lab to help develop a novel treatment, I immediately thought of Cure Rare Disease as a development partner. Given their experience in advancing novel therapeutics through lean development processes, Rich and his team are helping to greatly accelerate our preclinical development of gene therapy for this condition, and will be invaluable in moving from the laboratory to the clinic. This partnership is critical for moving the project forward without delay, in hopes of helping those impacted by the disease sooner rather than later.”

Diane Balderson, PhD

Regulatory Consultant

"From a regulatory perspective, the regulatory model that Cure Rare Disease is advancing for ultra-rare disease patients allows for the targeted preclinical testing of life-saving therapeutics. Cure Rare Disease's collaboration with the FDA to rationalize the drug development process for small groups of patients will help medicines for ultra-rare diseases develop into life-saving therapeutics."

Andelyn Biosciences

Manufacturing Partner

"Manufacturing gene therapies is one of the most challenging parts of the development process. The value of the CRD model to potentially help ultra-rare disease patients is incredible and we are proud to work closely with Cure Rare Disease to advance their mission."

Lou Kunkel, PhD

Director of Genomics Program, Harvard Medical School

"Having mentored and collaborated with Cure Rare Disease's Founder, Rich Horgan, for several years, it is awe-inspiring to see the evolution of the organization in such a short period of time. Cure Rare Disease has become a formidable research institution that is bringing cutting-edge technologies to patients who need them most."

Noah Weisleder, PhD

Professor & Director of Graduate Studies, Ohio State College of Medicine

"Having worked with Cure Rare Disease for the last few years to advance a novel protein therapeutic approach, I've found their collaborative and agile mindset to be a refreshing and effective force to advance innovative therapeutics into the clinic to help patients who urgently need them."

Ronald Cohn, MD

CEO, Hospital for Sick Children

“As a children’s hospital that sees patients who may be among only a few people in the world with their conditions, we share Cure Rare Disease’s urgency in the effort to develop drug development frameworks to treat rare diseases. We hope our work with Cure Rare Disease will help lead to new treatment avenues for patients where none existed before."

Steve Festin

Senior Director, Scientific & Commercial Development, Charles River

“Cure Rare Disease has developed an innovative framework of lean ultra-rare disease drug development paired with novel research model creation. The result helps bridge the gap between a drug's performance in an experimental setting and our expectations of its performance in a human patient. Moreover, CRD's willingness to share resources, such as novel research models, with a larger community benefits all drug development efforts and will make a marked difference for patients.”

Terence Flotte, MD

Provost & Executive Deputy Chancellor, UMass Chan Medical School

"Society is at a critical inflection point where we can not only diagnose but also potentially treat patients who are impacted by devastating rare diseases. Cure Rare Disease is at the forefront of this rapidly evolving paradigm. Their progress is intended to relieve the suffering of those patients and families who will be treated within the program and also on creating a societal benefit from the knowledge gained and shared that can be applied to other diseases."

Zhenya Ivakine, PhD

Scientist, Genetics & Genome Biology, Hospital for Sick Children

"The collaborative environment that Cure Rare Disease creates among its research collaborators is incredibly refreshing. CRD encourages scientists across disciplines and universities to work together with the goal of bringing real-life treatments to patients as soon as possible. This is uncommon in the drug development space but something that catalyzes great progress."

Willeke van Roon

Assistant Professor, Leiden University

"As the potential to treat even individual patients with advanced therapeutics grows, Cure Rare Disease is forging the path forward to develop a robust framework that society can leverage and learn from to treat other diseases. CRD’s collaborative model is a novel and more efficient way to approach drug development - not for profit, but rather to help patients with rare disorders that currently have no hope for an effective treatment."

Our Collaborators

Our Drug Development Model relies on our extensive network of academic and industry collaborations across the United States, Canada and Europe.

Let’s Talk

Interested in collaborating with Cure Rare Disease? Complete the form to connect with a member of our team.

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