CIRM’s Board received an update on our clinical portfolio, which now includes 60 trials across multiple diseases. The President’s Report highlights 15 trials, our Cure Sickle Cell Initiative with the NIH, and our educational programs.

We want to share key updates showing how our portfolio is maturing.

Clinical trials update

Many trials are now moving toward registration. Some are small Phase 2 or earlier trials designed to generate enough safety and efficacy data for FDA approval. Regenerative medicine often relies on small trials because patient populations are small, yet therapies can still show large effect sizes. CIRM programs often follow this model to address rare unmet needs.

Orchard Therapeutics is running a Phase 2 trial for ADA‑SCID, a rare immune disorder. They report long‑term immune recovery in 20 patients two years after treatment. Orchard plans to submit a Biologics License Application in 2020. Maria Millan said these results show strong progress and praised CIRM’s early support for the program. She noted that this success could open doors for thousands of untreated genetic diseases.

The trial uses gene therapy to correct the ADA gene in the patient’s own blood stem cells. ADA levels and new immune cells show the therapy is working. The program has received $19 million from CIRM and earned FDA Breakthrough Therapy and Orphan Drug designations.

LAD-I

Rocket Pharmaceuticals is testing a gene therapy for LAD‑1, a rare and fatal pediatric disorder. Early results from the first patient show safety and potential efficacy, including improvements in skin lesions. This small Phase 1 trial was designed to be registration‑enabling. Rocket hopes positive data will support discussions with the FDA about final approval. The program has received $6.6 million from CIRM.

CIRM also funds Forty Seven Inc. to run an immuno‑oncology trial for AML and MDS. The therapy blocks CD47, allowing immune cells to kill cancer stem cells. It is paired with chemotherapy to improve effectiveness. The trial has FDA Fast Track and Orphan Drug status and has received $5 million from CIRM.

Other registration‑phase trials include several Phase 3 programs:

Brainstorm Cell Therapeutics is testing a therapy for ALS. Enrollment is complete, and results are expected in late 2020.

Humacyte is developing bioengineered vessels for hemodialysis access. These Phase 3 trials compare bioengineered vessels to synthetic grafts and patient vessels. Humacyte received the first FDA RMAT designation in 2017 and has received $24 million from CIRM.

Medeor Therapeutics is running a Phase 3 trial combining blood stem cell and kidney transplantation to induce immune tolerance. The goal is to eliminate the need for lifelong immunosuppressive drugs. CIRM has provided $11.2 million in funding.