Cure Rare Disease

03/18/2026

Families facing ultra-rare diseases often fight their battles in the shadows. We are deeply grateful to Becky Quick and CNBC for shining a light on these journeys through CNBC Cures, giving a crucial voice to the rare disease community and the innovators working to find solutions.

Tune in tomorrow night, Thursday, March 19, at 7 p.m. EST for the premiere of “Defying Rare Disease” on CNBC.

Our Founder and CEO, Rich Horgan, sits down with Becky to discuss how we develop genetic treatments for ultra-rare conditions, the roadblocks the biotechnology industry must still overcome, and the enduring legacy of his brother, Terry, which fuels our mission every day.

03/02/2026

We are proud to announce our partnership with the LGMD2L Foundation to develop a novel gene replacement therapy for Anoctamin 5 (ANO5)-related disease, a rare genetic disorder.

The transformational partnership will fund a comprehensive, multi-year program dedicated to advancing a potential treatment from initial design to clinical trial readiness. The funding provided by the LGMD2L Foundation will support a series of critical milestones, including therapeutic design, preclinical studies, manufacturing scale-up, and ultimately a first-in-human clinical trial.

Learn more: http://bit.ly/4rNhO29

12/16/2025

We are thrilled to announce Cure Rare Disease has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine to advance our gene therapy program, CRD-003, for Limb-Girdle Muscular Dystrophy Type 2i/R9.

This funding is designated to support critical next steps in the program's development:

- Completion of late-stage preclinical development.
- Initiation of GMP manufacturing for the drug candidate.
- Submission of an Investigational New Drug (IND) application to the FDA.
- Launch of a Phase 1/2 clinical trial.

The therapy utilizes a novel, liver-detargeting, muscle-tropic AAVMYO2 capsid intended to improve safety and efficacy. We appreciate CIRM's commitment to this program as we work to bring a therapeutic candidate forward for the LGMD2i/R9 community.

Read more here: https://www.cureraredisease.org/blog-posts/cure-rare-disease-awarded-7-4-million-cirm-grant-to-advance-gene-therapy-for-limb-girdle-muscular-dystrophy-type-2i-r9

12/02/2025

"I know what it means to stand in that place."

That place is where a doctor says nothing can be done in the wake of a devastating diagnosis.

On Giving Tuesday, our Chief Advancement Officer, Brittany Stineman, shares her experience hearing these words after her son, Nash, was diagnosed with a fatal, ultra-rare disease. She rejected the verdict and, through sheer grit, led the charge to develop the first-ever gene therapy for SMARD1.

That powerful determination is the heart of Cure Rare Disease. We lift that crushing weight off families. We transform isolation into collaboration, despair into direction, and heartbreak into breakthroughs that were once unimaginable. We develop life-changing treatments for patients once deemed "too rare to treat."
And your support fuels our progress.

In 2025 alone, your support enabled milestones like Orphan Drug Designation for our LGMD2i and SCA3 therapies, the development of next-generation AAV vectors for CRISPR-based Duchenne muscular dystrophy treatments, and more.

Every discovery, every regulatory step, brings us closer to clinical trials.

Read Brittany's full, inspiring letter on how CRD is pioneering breakthroughs — and then help us accelerate the science.

This year, I had the profound honor of joining the Cure Rare Disease (CRD) team as Chief Advancement Officer. Every day, I stand alongside families and patients who have heard the most devastating words a doctor can deliver, and still refuse to surrender. Families who reject the idea that “nothing...

09/26/2025

We are proud to share that the U.S. Food and Drug Administration has granted Orphan Drug Designation to our investigational gene therapy, CRD-003, for the treatment of Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9).

“Receiving Orphan Drug Designation is a critical milestone for Cure Rare Disease as we advance our gene therapy program for LGMDR9,” said Richard Horgan, Founder and CEO of Cure Rare Disease. “Patients and families affected by FKRP-related muscular dystrophies currently face a devastating lack of treatment options. This designation not only supports our approach, but also underscores the urgent need for innovative genetic medicines that can meaningfully change the lives of patients and their loved ones. Using a liver de-targeting, second-generation AAV vector, we believe this may be a safer approach compared to first-generation capsids.”

Learn more: http://bit.ly/3IFDrjw

07/10/2025

The new Cure Rare Disease Annual Report is out now!

Read the annual report in full for a detailed look at our most recent advancements, program updates, and more — all possible because of commitment and dedication from our research collaborators, industry partners, board members, families, community and staff.

Download the full report here: https://shorturl.at/voJKI