Stargardt Disease

Stargardt Disease

Stargardt disease is an inherited eye disease that causes progressive loss of vision. It specifically affects the central part of the retina, known as the macula. Although Stargardt disease is the most common inherited disease of the macula, it’s still considered rare. According to the Foundation Fighting Blindness, an estimated 30,000 people are affected in the United States.

Gene

ABCA4

Disease-causing Mutations

1,000+

Cause

Mutations, or “mistakes,” in both copies of a person’s ABCA4 gene are the most common cause of Stargardt disease. Usually, these mutations are inherited, one from each parent (the parent will not have Stargardt if they only have one copy of the mutation.) ABCA4 is a large gene responsible for making a protein involved in transporting toxins (vitamin A molecules) out of cells. When there is a defect in this gene, the protein is not produced or does not work correctly, leading to the death of retina cells and progressive central vision loss. More than 1,000 different mutations in ABCA4 can lead to Stargardt disease.

Disease Progression

The first sign of vision loss usually occurs in children – but some people don’t start to lose their vision until they’re adults. In the early stages, people may encounter challenges when reading or seeing in low-light situations, have trouble adapting to changes in light and darkness, or experience vision impairments like blurriness or distortion.

While the progression and severity of Stargardt disease can vary, those in the advanced stages will have trouble distinguishing details and shapes, making simple tasks like reading, driving, and recognizing faces challenging or impossible.

Treatment

Vision loss from Stargardt disease can’t be fixed with glasses, contact lenses, or surgery, and no FDA-approved treatment is available – we aim to change that. Using our novel Gene Coding™ technology, the SalioGen team is developing a genetic medicine that can integrate a healthy copy of the ABCA4 gene into the retina to slow or stop the progressive loss of central vision in people with Stargardt disease – regardless of mutation. We are working urgently to advance this potential one-time, non-viral therapy into its first-in-human clinical trial in 2025.

Helpful Resources

Below are organizations that offer resources and information for patients, families, and caregivers living with Stargardt disease. This list is provided for reference only and is not intended as a comprehensive list of resources.

Foundation Fighting Blindness

View resource

The Carroll Center for the Blind

View resource

National Eye Institute

View resource

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    Kalliopi “Kali” Stasi, M.D., Ph.D.

    Chief Medical Officer

    Kali is a board-certified ophthalmologist with 30 years of experience in industry and academia, and a proven track record of advancing genetic medicines for retinal diseases from early- and late-stage clinical development through commercialization.

    Kali most recently served as the Senior Vice President of Clinical Development at Adverum Biotechnologies responsible for planning and executing the company’s global clinical development programs for vision loss. In her previous position, Kali led Tenpoint Therapeutic’s cell therapy program for ophthalmic indications as Senior Vice President of Clinical Development. Before that, Kali worked at Novartis Institutes for Biomedical Research as a Translational Medical Director, where she led multiple clinical trials in a pipeline of gene and cell therapies intended to treat retinal diseases. Earlier in her career, Kali served four years at the University of Pennsylvania as an Assistant Professor of Ophthalmology.

    Kali earned her M.D. and Ph.D. in neurodegenerative disease from the University of Patras, Greece. She completed an ophthalmology residency at the University of Rochester, NY, a Cornea and Refractive Surgery Clinical fellowship at Johns Hopkins Wilmer Eye Institute, and a business post-baccalaureate program at Wharton Business School.

    Jason F. Cole

    CEO & Chairman of the Board

    Jason is a strategic biotechnology CEO and board member with over 20 years of experience working with public and private biotech companies focused on strategy, capital raising, business development, operations, and enabling teams.

    Jason joined SalioGen from bluebird bio, where he served as Chief Strategy and Financial Officer, Chief Business Officer, and Chief Operating and Legal Officer, providing strategic, financial, operational, and legal oversight during key growth and transition periods. In these roles, bluebird bio scaled from 100 to 1,200 employees (U.S. & Europe), secured FDA approval of three first-of-their-kind gene and cell therapies (ZYNTEGLO, SKYSONA, ABECMA), and closed over $3.7 billion in financings and strategy-enabling business development transactions. Before bluebird bio, Jason held positions on the executive leadership teams of Zalicus and CombinatoRx, with responsibility across corporate development, strategic alliances, legal, IT, and facilities.

    Jason earned a J.D. from Columbia University and an A.B. in Government from Dartmouth College.

    Joseph J. Higgins, M.D.

    Chief Innovation Officer, Co-Founder & Board Member

    Joe is an executive leader, clinician, and scientist dedicated to advancing gene therapies for children and adults with genetic disorders involving the cardiovascular, ophthalmological, neurological, and immunological systems. His background spans pediatrics, child neurology, medical genetics, and molecular developmental neurobiology.

    Earlier in his career, Joe worked on the Human Genome Project as the lead of the Human Genetic Initiative. His academic background includes positions as an instructor at Harvard Medical School, an Assistant Professor at George Washington University, an Associate Professor at SUNY Albany, and a Professor at Cornell University. As the Vice President of Clinical Development at uniQure, Joe led Phase 1/2 first-in-human gene therapy trials in Huntington’s disease.

    Joe received his medical degree from New York University and graduated cum laude with a B.A. in Biology from Marist College. He is double-board certified in Pediatrics and Neurology with Special Qualification in Child Neurology. He is licensed to practice medicine in New York and Massachusetts.

    Jeff Kopacz, M.S., J.D.

    Senior Vice President, Intellectual Property & Contracts

    Jeff has extensive intellectual property (IP) experience, including building and defending IP protection for platform-based technologies and FDA-approved therapeutics.

    Previously, Jeff served as Vice President of Intellectual Property at Omega Therapeutics, leading IP and contract activities related to their genomic control technology. Before Omega, Jeff worked at Alnylam Pharmaceuticals for ten years, most recently as Senior Director, Senior Intellectual Property counsel.

    Earlier, Jeff was an attorney at Edwards Angell Palmer & Dodge LLP (now Locke Lord LLP) Jeff began his biopharma career at Millennium Pharmaceuticals (now Takeda Pharmaceuticals).

    Jeff received his undergraduate degree in microbiology from the University of Massachusetts at Amherst, his graduate degree in molecular microbiology and immunology from Johns Hopkins University, and a law degree from Suffolk University Law School.

    Kate Lewis

    Vice President, Portfolio and Program Management

    Kate is a biotech leader with 20 years of experience in life sciences and drug development, including extensive work in gene therapy. Her focus is portfolio management, strategic planning, communications, and team leadership.

    Prior to SalioGen, Kate served as Vice President, Program Lead and Head of Program Management at Syndax Pharmaceuticals and Freeline Therapeutics. Before that, Kate spent nearly seven years at bluebird bio in roles in Medical Affairs and Program Leadership.

    Kate holds a B.A. from Pomona College and an M.A. from the University of Iowa, both in English literature.

    Will McGowan

    Executive Director, Finance & FP&A

    Will is a financial leader with over 15 years of experience working in both public and private biotech companies at all stages of development, from discovery through commercialization.

    Will most recently was the Senior Director of FP&A for Allena Pharmaceuticals. Before that role, He spent five years as the Senior Director of Finance and Controller for Navitor Pharmaceuticals and nine years at AMAG Pharmaceuticals in various roles, including Controller and Director of FP&A.

    Will holds an MBA from Quinnipiac University and a Bachelor of Science in Finance and Accounting from the Darla Moore School of Business at the University of South Carolina.

    Jill Murray

    Vice President, Human Resources

    Jill is a senior human resources professional with 20+ years of progressive experience in the biotech industry. Her focus is organizational design, employee development, employee relations, leadership coaching, change management, compensation, benefits administration, and legal compliance.

    Jill began her career at ImmunoGen, where she covered all facets of Human Resources and Talent Management during her 14-year tenure. After that, Jill joined Sarepta Therapeutics, where she was responsible for establishing and building the HR Business Partnering function, followed by Rubius Therapeutics where she served as Head of Human Resources.

    Jill holds a bachelor’s degree in art and psychology from Carnegie Mellon University and is a certified Executive Coach.

    Sandeep Nema, Ph.D.

    Senior Vice President, Technical Operations

    Sandeep has extensive developmental experience in bringing candidates to clinic that span various modalities, including gene therapy, ADCs, mAbs, vaccines, oligonucleotides, bi/tri-specific, and CART.

    Before SalioGen, Sandeep worked for 24 years at Pfizer. Recently, his team successfully brought Pfizer’s COVID-19 LNP-mRNA vaccine to the clinic and achieved emergency use authorization. In his last role as Executive Director, Biotherapeutics Pharmaceutical Sciences, Global Biologics, Sandeep was responsible for all pre-proof-of-concept biologics projects, approximately 50 in total, from discovery up to Phase 3 start. Before that, Sandeep established the Protein Pharmaceutical R&D group at Pfizer, which included Formulation, Microbiology, and Stability functions. He is also an adjunct Professor at the University of Tennessee.

    Sandeep earned his Ph.D. in Pharmaceuticals from the University of Tennessee Health Science Center and his Bachelor of Pharmacy from Banaras Hindu University. He is also a Certified Regulatory Affairs Professional.

    Joe Senn, Ph.D.

    Senior Vice President, Head of R&D

    Joe is an R&D leader with experience in nearly all therapeutic modalities, including small molecules, biologics, antisense, gene editing, and mRNA therapeutics. He most recently served as the Vice President of Nonclinical Development at Moderna Therapeutics, where he and his team were responsible for progressing over 40 candidates into the clinic. Before Moderna, Joe served as Site Head for Drug Safety at Takeda Pharmaceuticals, overseeing the development of all immunology products across the portfolio.

    Joe earned his Ph.D. in Pharmacology and Physiology from the University of Rochester School of Medicine and Dentistry and his B.S. from Niagara University.