- SB-007 is the only IND-cleared, clinical-stage therapeutic
addressing the root cause of Stargardt disease with the potential
to treat all patients across all ABCA4 mutations
- Phase 1/2 ASTRA study set to begin in the first half of
2025, supported by POLARIS, a pioneering company-sponsored natural
history study in Stargardt disease
- First ever IND clearance for a Protein Splicing gene
therapy
BARCELONA, Spain, Dec. 12,
2024 /PRNewswire/ -- SpliceBio, a genetic medicines
company pioneering Protein Splicing to address diseases caused by
mutations in large genes, today announced that the U.S. Food &
Drug Administration (FDA) has cleared its investigational new drug
(IND) application for lead program SB-007. SB-007 is the only
clinical-stage therapeutic addressing the root genetic cause of
Stargardt disease with the potential to treat all patients across
all ABCA4 mutations.
Miquel Vila-Perelló, Ph.D., Chief Executive Officer, and
Co-Founder of SpliceBio, said: "The FDA IND clearance of
SB-007 is a significant achievement for SpliceBio and Stargardt
disease patients. As the first-ever IND for a Protein Splicing gene
therapy, it is a huge step forward to demonstrate the potential of
this new therapeutic modality to address diseases caused by
mutations in large genes such as ABCA4. SB-007 is an
adeno-associated viral (AAV) vector gene therapy aimed at restoring
expression of the full-length ABCA4 protein, and the only
clinical-stage therapy with the potential to help all Stargardt
patients. We look forward to accelerating the clinical development
of SB-007, building on the Orphan Drug Designation granted by the
FDA in 2024, and advancing this potentially life-changing
therapeutic for patients with Stargardt disease."
"Stargardt disease has been a challenge for the development of
gene therapies due to the large size of
the ABCA4 gene, and currently has no approved
therapies available," said Professor Paul Yang, M.D., Ph.D., Chief of the Paul H.
Casey Ophthalmic Genetics Division at Casey Eye Institute at Oregon
Health & Science University. "This new
therapy utilizes a unique approach to replace the full-size,
normal ABCA4 protein at high efficiency, which
addresses the root cause of Stargardt disease across any pathogenic
variant in the ABCA4 gene. This IND clearance
represents a major milestone in the field and I am thrilled to be
part of the clinical studies exploring this promising approach that
could transform the lives of Stargardt disease patients."
SpliceBio plans to initiate enrolment in the Phase 1/2 ASTRA
study in the first half of 2025. ASTRA will evaluate the safety and
efficacy of a single dose of SB-007 administered subretinally in
patients with Stargardt disease. In March
2024, SpliceBio launched the POLARIS trial, a pioneering
company-sponsored natural history study of Stargardt disease
designed to evaluate disease progression, refine endpoints, and
streamline eligibility criteria for accelerated enrolment into
the Phase 1/2 ASTRA study. This study will enable Stargardt disease
patients to benefit from more precise diagnoses, more rigorous
disease monitoring, and potentially faster access to innovative
therapies.
The SpliceBio Management team will be attending J.P. Morgan's
43rd Annual Healthcare Conference 2025, being held in San Francisco from 13-16 January 2025. Please get in contact to
schedule a meeting at bd@splice.bio.
About Stargardt disease
Stargardt disease is the most common form of inherited juvenile
macular degeneration, affecting approximately 1 in 8,000 to 10,000
individuals. Caused by mutations in the ABCA4 gene,
Stargardt disease leads to the progressive loss of central vision
due to damage to the central region of the retina known as the
macula. The disease is variable in the age of onset, including
early onset in children and adolescents and late-onset forms in
adulthood. There are no approved treatments, and patients face
significant challenges in daily life as the disease progresses.
Stargardt disease has remained elusive to genetic medicines due to
the large size of the ABCA4 gene.
About SB-007
SB-007 is a Protein Splicing dual AAV gene therapy that delivers
the full-length ABCA4 gene and aims to restore expression of the
native ABCA4 protein in the retina. It has demonstrated robust
pharmacological activity in animal models of Stargardt disease and
durable expression and safety in non-human primates. SB-007 has
been granted Orphan Drug Designation by the FDA in the US and the
European Commission in Europe.
About SpliceBio
SpliceBio is a genetic medicines company pioneering Protein
Splicing to address diseases caused by mutations in large
genes. The Company's lead program, SB-007, targets the root
cause of Stargardt disease, a genetic eye disease that causes
blindness in children and adults. SpliceBio's pipeline comprises
additional gene therapy programs across therapeutic areas,
including ophthalmology and neurology. SpliceBio's platform is
based on technology developed in the Muir Lab at Princeton University after more than 20 years of
pioneering intein, Protein Splicing, and protein engineering
research. For additional information, please visit
www.splice.bio.
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SOURCE SpliceBio