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Advancing Ex Vivo Gene Therapy: IND-Enabling Studies for ASAH1-related SMA-PME

Award Number: U01NS151207
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/18/2026
PERIOD OF PERFORMANCE END DATE: 08/31/2028

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Advancing Ex Vivo Gene Therapy: IND-Enabling Studies for ASAH1-related SMA-PME - Project Summary / Abstract ASAH1-related Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA-PME) is an ultra-rare neurogenetic disorder predominantly affecting adolescent children. ASAH1 encodes acid ceramidase, an enzyme involved in sphingolipid metabolism. Its activity is crucial for maintaining the balance of bioactive lipids and cellular homeostasis. A partial deficiency results in a progressive neuropathology marked by seizures (progressive myoclonic epilepsy, PME) and muscle weakness (spinal muscular atrophy, SMA), often leading to death within 5 to 10 years of diagnosis. There is currently no treatment for this condition. By utilizing autologous hematopoietic stem/progenitor cells (HSPCs) genetically engineered to overexpress human acid ceramidase, we aim to deliver a safe and effective single-dose treatment that could potentially offer a lifetime of enzyme replacement. This approach of lentiviral vector-mediated ex vivo gene therapy has demonstrated safety and efficacy in a variety of human diseases, transforming the treatment landscape for conditions once considered incurable. Extensive preclinical studies of safety, efficacy and biodistribution using our proposed drug product have been shown by our collaborator and are outlined in the proposal. Additional animal model studies are not required based on feedback from the FDA delegates who reviewed our Pre-IND briefing, prompting our advancement to request this funding mechanism for IND-enabling support. Our proposed clinical trial will utilize a low-intensity conditioning regimen that is well-tolerated and effective for HSPC repopulation. At Cedars-Sinai, we possess the expertise and infrastructure to execute the entire process for this proposed therapy from start to finish. We have a world-class hospital with expertise in apheresis and transfusion for adults and children. Our Cell Biomanufacturing Center is a state-of-the-art facility that provides the infrastructure and expertise needed to produce clinical-grade cellular products, and they foster close collaboration with internal investigators to translate discoveries into clinical therapies. We will establish a comprehensive Good Manufacturing Practice (GMP) process to produce this cellular treatment at scale, collect the necessary Chemistry, Manufacturing, and Controls (CMC) data, and develop detailed clinical trial plans. This will enable us to submit a complete Investigational New Drug (IND) package for expedited review by the FDA and provide new hope to the affected children and their families.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $825,159 )
2026	2026	CEDARS-SINAI MEDICAL CENTER	8700 BEVERLY BLVD	WEST HOLLYWOOD	CA	90048	LOS ANGELES	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	9/17/2026	NEW	$825,159
														Subtotal = $825,159

Grand Total All Awards = $825,159

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Advancing Ex Vivo Gene Therapy: IND-Enabling Studies for ASAH1-related SMA-PME

Award Number: U01NS151207

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/18/2026

PERIOD OF PERFORMANCE END DATE: 08/31/2028

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