Abeona Expands Pipeline with Addition of Cystic Fibrosis Gene Therapy

December 6, 2018

Rare Daily Staff

Abeona Therapeutics said it expanded its pipeline with the addition of ABO-401, an experimental gene therapy for cystic fibrosis that corrects the underlying deficit in the rare lung disease regardless of a patient’s specific mutation.

Cystic fibrosis is a rare, life-shortening genetic disease. It is caused by a defective or missing cystic fibrosis transmembrane conductance regulator, or CFTR protein. There are approximately 2,000 known mutations in the CFTR gene. The mutations that drive the disease create non-working or too few CFTR proteins at the cell surface.

Data the company presented during its R&D Day in New York City suggest that ABO-401, based on the vector AAV204, efficiently targets lung cells and that ABO-401 corrects the underlying cystic fibrosis chloride channel deficit. ABO-401 is the first program based on the AIM vector platform for gene therapy delivery. AIM is the next-generation gene therapy AAV capsid portfolio Abeona licensed from the University of North Carolina at Chapel Hill.

Unlike earlier AAV capsids used to carry genetic material, the AIM vector platform is capable of widespread central nervous system gene transfer and can be used to effectively deliver therapies for various indications. Studies indicate that AIM vectors can efficiently and broadly target CNS tissue, and may provide a treatment for patients that have inhibitory antibodies to natural AAV serotypes. Importantly, the AIM vector system may provide second-generation treatment approaches for patients that have received a previous AAV injection.

“Our AIM vector platform enables the potential for gene therapy for patients living with cystic fibrosis, regardless of mutation, which could change the landscape of treatment and alter the course of this progressive, genetic disease,” said Timothy Miller, co-founder, president, and chief scientific officer. “We are very encouraged by the preclinical data presented today demonstrating delivery and correction of the underlying genetic deficit in CF patient cells.”

Miller said the company showed AIM vectors could deliver genes to the eye and are excited about their potential as the next generation of gene therapy across tissue types.

The company also provided updates on other programs. Abeona said it expects to initiate a pivotal study in the middle of 2019 for EB-101, its experimental gene-corrected cell therapy to treat recessive dystrophic epidermolysis bullosa, a skin disease characterized by chronic epidermal wounds in which patients suffer from pain, itching, and widespread complications impacting quality-of-life and life expectancy.

Abeona plans to amend its ongoing phase 1/2 study of ABO-102, its gene therapy for MPS IIIA, a lysosomal storage disease with no approved treatment that is characterized by neurodevelopmental decline, behavior abnormalities, seizures, loss of speech or vision, an inability to sleep, and premature death. The company is amending the study to enroll patients at earlier stages of disease. The study has demonstrated a substantial, dose-related improvement in biomarkers, including reductions in cerebrospinal fluid heparan sulfate levels and liver volume in patients treated with ABO-102. Investigators also observed encouraging neurocognitive signals in younger, higher functioning patients enrolled in the higher dose of cohort.

The company also presented preclinical data on ABO-202 for the treatment of CLN1, a rare and fatal autosomal recessive genetic disorder with no approved treatment, which is characterized by vision impairment and rapid neurological regression. Abeona expects to file an application in the first quarter of 2019 to begin human studies.

December 6, 2018
Photo: Timothy Miller, co-founder, president, and chief scientific officer of Abeona

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