CLEVELAND, Ohio –Sangrail Biologics, a clinical-stage gene therapy company focused on restoring life’s blueprint for children with rare genetic diseases, announced the launch of the company today and highlighted the company’s lead clinical asset, SNG-101 (f.k.a. ABO-101), a potential first-in-class AAV gene therapy designed to treat the underlying cause of disease in children with mucopolysaccharidosis type IIIB (MPS IIIB, a.k.a. Sanfilippo syndrome type B).
“The launch of Sangrail Biologics is driven by a singular, urgent conviction: that we can fundamentally change a child’s trajectory after diagnosis with a rare, neurodegenerative disease. Our mission of restoring life’s blueprint is a promise we are making to the families whose children are fighting battles against conditions that currently have no cure,” said CEO, Chairman and Co-Founder Timothy J. Miller, Ph.D. “We are continuing our journey with SNG-101 for patients with MPS IIIB and applying the precision of AAV gene therapy to address the root cause of the disease at the cellular level. We are here to transform these diagnoses from a path of decline into a future defined by biological restoration, potential, and hope.”
SNG-101 is an adeno-associated viral type 9 (AAV9) gene therapy delivered by a single intravenous injection for children with MPS IIIB, a rare autosomal recessive disease, causing neurocognitive decline, speech and mobility loss, and premature death. SNG-101 introduces a correct copy of the NAGLU gene that causes MPS IIIB disease to restore correct enzyme function and enable the breakdown of the toxic accumulation of heparan sulfate in cells, particularly in the central nervous system (CNS). The company announced that 14 MPS IIIB patients have received SNG-101 through a global, open-label, dose escalating Phase 1/2 clinical trial and named-patient programs. Patients received SNG-101 through an intravenous delivery at 2E13 vg/kg (n=2), 5E13 vg/kg (n=5), or 1E14 vg/kg (n=7).
“MPS IIIB is a devastating and progressive lysosomal storage disease with few treatment options,” stated Kevin M. Flanigan, MD, principal investigator and Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We see sustained reductions in several key biopotency biomarkers, including significant reductions in heparan sulfate in the cerebral spinal fluid, urine and plasma, normalization of plasma NAGLU enzyme activity, and diminished liver volume as measured by MRI through 18-24 months of follow up. Importantly, we also see improvements or stabilization of neurocognitive function in the youngest treated patients.”
The results from the global Phase 1/2 clinical trial will be presented on Wednesday May 13 at 3:30 PM EST at the annual meeting of the American Society of Gene and Cell Therapy, held this year in Boston, MA.
In the 11 patients treated in the Phase 1/2 clinical study, all CSF, plasma and urine biomarkers improved significantly over the duration of the study (p<0.01). NAGLU enzyme activity in the CSF showed a dose-dependent response, normalizing in all subjects by 30 days and remained within normal limits during the entirety of the study, based on natural history comparators. NAGLU enzyme activity in plasma normalized in all subjects by 7 days and remained higher than baseline throughout the study. GM2 and GM3, which are biomarkers of neuronal loss, decreased in the CSF by 64% and 50% respectively, while glycosaminoglycans (GAG)s in urine decreased by 67%. Liver and spleen volumes (adjusted for weight and height) significantly decreased as early as 6 months and remained decreased throughout the study (p<0.001).
“We are profoundly grateful to the families whose commitment to this program and long-term follow-up has been the backbone of our progress. This has truly been a community effort,” said Michelle Berg, President, COO and Co-Founder. “Advancing this program fulfills a deep responsibility to the families who have been waiting for a solution that addresses the genetic root of MPS IIIB. We are committed to turning this research into tangible hope, believing that children with this diagnosis should have the opportunity to develop without limitations.”
Sangrail’s MPS IIIB program, SNG-101, has secured robust regulatory support across major markets. In the United States, the program holds FDA Fast Track and Orphan Drug designations, alongside a Rare Pediatric Disease Designation that qualifies SNG-101 for a Priority Review Voucher (PRV) upon BLA approval. Internationally, the program has also been granted Orphan Drug Designation by the European Medicines Agency (EMA).
“Similar to clinical studies for patients with MPS IIIA, the strength of the SNG-101 clinical data generated over these years of follow-up tells a compelling story of potential and rigorous scientific achievement,” stated Terri Klein, CEO of the USA National MPS Society, USA. “As we represent families who navigate these rare genetic challenges every day, the Society recognizes the vital importance of translating robust, long-term clinical evidence demonstrated by SNG-101 into real-world availability for patients with MPS IIIB. We fully support the efforts to accelerate access to this gene therapy, as it represents exactly the kind of breakthrough progress our community has been fighting for.”
About SNG-101 (f.k.a ABO-101): ABO-101 was originally a program from Abeona Therapeutics, which licensed the program from Nationwide Children’s Hospital (Columbus, OH), and advanced the program from pre-clinical evaluation to a first-in-human clinical trial. SNG-101 is a potential first-in-class, systemically administered, adeno-associated viral (AAV)-based gene therapy designed to treat the underlying cause of disease in children with mucopolysaccharidosis type IIIB (MPS IIIB, a.k.a. Sanfilippo syndrome type B). Treatment involves a one-time, single, intravenous delivery of a functioning copy of the N-acetyl-α-D-glucosaminidase (NAGLU) gene to cells of the central nervous system (CNS) and peripheral organs, with the aim of correcting the effects that result from the genetic aberrations that are the root cause of the disease. Clinical studies supporting the use of AAV9 gene therapy to treat patients with Sanfilippo syndrome have been published in several peer-reviewed scientific journals. Clinically, fourteen (14) patients with MPS IIIB have received SNG-101 through a global, open-label, dose escalating Phase 1/2 clinical trial and named-patient programs.
About MPS IIIB: (also known as Sanfilippo syndrome type B) is a genetic, progressive, and devastating rare lysosomal storage disease. In patients with MPS IIIB, genetic mutations result in a marked decrease in NAGLU enzyme activity, which leads to accumulation of heparan sulfate (HS) in the cells in the brain and other organs as well as progressive brain atrophy with cortical gray matter volume loss. The accumulation of abnormal HS in cells is toxic, and results in neurocognitive decline, behavioral disturbances, speech loss, increasing loss of mobility, and premature death. MPS IIIB typically presents in children during the first few years of life, and 70% of patients do not reach 18 years of age. There are no approved treatments for MPS IIIB.
About Sangrail Bio: Sangrail Biologics Inc. is a clinical-stage gene therapy company with a mission of restoring life’s blueprint for children born with rare genetic diseases. The company’s lead program, SNG-101 (f.k.a. ABO-101) is a late-stage AAV9-based gene therapy clinical program for patients Sanfilippo syndrome type B (MPS IIIB).
For more information, visit https://sangrailbiologics.com
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For patients and families living with MPS IIIB – [email protected]
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