Kamau Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for Nula-cel, a Gene-Corrected Stem Cell Therapy for Sickle Cell Disease

Designation based on preliminary clinical evidence from the Phase 1/2 RESTORE trial; nula-cel also holds Fast Track and Orphan Drug designations

SOUTH SAN FRANCISCO, Calif., Nov. 12, 2026 (GLOBE NEWSWIRE) -- Kamau Therapeutics, a clinical-stage company creating medicines that endure by precisely engineering patients’ own blood stem cells, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nula-cel, an investigational stem cell medicine for sickle cell disease. The designation is based on preliminary clinical evidence from the ongoing Phase 1/2 RESTORE trial.

In RESTORE, all three patients treated with the optimized manufacturing process achieved transfusion independence with no vaso-occlusive events. The two patients with more than six months of follow-up have sickle hemoglobin below 20%, under the 30% threshold targeted for stroke prevention, with a predominantly adult hemoglobin profile.

RMAT designation is granted when preliminary clinical evidence indicates that a regenerative medicine therapy has the potential to address unmet medical needs in a serious condition. Designated therapies receive early, frequent FDA engagement and may be eligible for priority review and accelerated approval.

“Pain crises matter, but between them, sickled cells continue damaging the body. That is why guidelines call for exchange transfusions to keep sickle hemoglobin below 30% to prevent stroke,” said Vali Barsan, MD, Chief Executive Officer of Kamau Therapeutics. “We designed nula-cel to correct the root cause of sickle cell disease in a single treatment, and our team’s tireless work across research, manufacturing, quality, and clinical development has brought it into the clinic. This designation recognizes early clinical evidence of that correction as we advance nula-cel towards more patients.”

“RMAT designation grants us enhanced, more frequent interactions with the FDA as we plan the next stage of clinical development,” added Jacob Garcia, MD, Chief Medical Officer of Kamau Therapeutics. “We plan to request a comprehensive multidisciplinary RMAT meeting the agency recommended to align on the path to a registrational dataset, including the potential for priority review and endpoints for accelerated approval. We look forward to sharing additional interim efficacy data from RESTORE later this year.”

About Nula-cel
Nula-cel is an investigational stem cell medicine for sickle cell disease. Rather than inducing fetal hemoglobin to compensate for the disease, nula-cel is designed to correct the sickle mutation itself, converting it to the normal adult β-globin sequence. Using ex vivo homology-directed repair (HDR), Kamau precisely engineers a patient’s own long-lived stem cells outside the body, rigorously verifying each cell product’s quality, identity, potency, and safety before it is returned to the patient, a level of control not possible when editing occurs inside the body. As these cells self-renew, a one-time treatment is designed to deliver durable, potentially lifelong benefit. In the ongoing Phase 1/2 RESTORE trial (NCT04819841), nula-cel has shown transfusion independence and no vaso-occlusive events in all three patients treated with the optimized manufacturing process, and sickle hemoglobin below 20% with a predominantly adult hemoglobin profile in the two patients with more than six months of follow-up. Nula-cel has received RMAT, Fast Track, and Orphan Drug designations from the FDA.

About Sickle Cell Disease
Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 100,000 people in the United States and millions globally. A single mutation in the β-globin gene produces sickle hemoglobin (HgbS), which polymerizes, stiffening red cells and driving chronic hemolysis. Pain crises are the visible burden; chronic organ damage, including stroke and injury to the kidneys, retinas, and bones, accumulates silently between them, shortening U.S. life expectancy by decades. While approved pharmacologic and gene therapies reduce pain crises, none restores native adult hemoglobin (HgbA) or consistently achieves the sustained HgbS levels below 30% that ASH guidelines associate with stroke prevention in chronic exchange transfusion protocols.

About Kamau Therapeutics
Kamau Therapeutics is a clinical-stage company developing a new generation of stem cell medicines designed to create lasting benefit through a single intervention. Kamau’s platform precisely engineers long-lived stem cells outside the body, providing control to select, modify, characterize, and verify cells before they return to the patient. As these cells self-renew and continually produce new cells, a change made once has the potential to endure. Kamau’s approach is built on homology-directed repair (HDR), the high-fidelity pathway cells naturally use to repair their own DNA, which Kamau has engineered to be efficient and reproducible in hematopoietic stem cells. Kamau’s lead program, nula-cel, is an investigational therapy for sickle cell disease designed to directly correct the sickle-causing mutation at its native location in the β-globin gene, restoring production of adult hemoglobin under the body’s own regulation rather than relying on fetal hemoglobin. Kamau’s integrated, cross-functional team, many of whom have advanced nula-cel from bench to bedside, is united by a single purpose: delivering stem cell medicines to patients. Learn more at kamautx.com.

Company Contact
info@kamautx.com

Media Contact
Michael Fitzhugh
LifeSci Communications
mfitzhugh@lifescicomms.com