SalioGen Therapeutics Announces Selection of Development Candidate for ABCA4-mediated Stargardt Disease

Lexington, Mass.

SGT-1001 is the first development candidate using SalioGen’s novel Gene CodingTM technology for gene integration via transposition

SalioGen plans to advance SGT-1001 into the clinic in the first half of 2025

Preclinical data for SGT-1001 will be presented at the ARVO and ASGCT annual meetings

Lexington, Mass., April 17, 2024 — SalioGen Therapeutics, a biotechnology company developing next-generation genetic medicines based on its novel Gene CodingTM technology, today announced the nomination of a development candidate, SGT-1001, for the treatment of Stargardt disease, a rare and progressive inherited retinal disease. SGT-1001 is being developed as a one-time, non-viral therapy to slow or stop the progressive loss of central vision in people with Stargardt disease, regardless of the type of mutation.

The SGT-1001 development candidate is the first to use SalioGen’s novel Gene Coding technology. Gene Coding integrates large or multiple whole genes into the genome at precise locations through transposition. This mechanism does not induce double-strand breaks or use guide RNA and has the potential to overcome the safety risks and limitations of other genetic therapeutic approaches.

“The nomination of SalioGen’s first development candidate, SGT-1001, demonstrates many of the unique capabilities of our novel Gene Coding technology, including the ability to seamlessly integrate a gene as large as ABCA4 into the retina with a proprietary lipid nanoparticle,” said Jason Cole, CEO of SalioGen Therapeutics. “This milestone is an important step forward for SalioGen and for people living with Stargardt disease who currently have no treatment options. We look forward to completing IND-enabling studies for SGT-1001 in the second half of 2024 and aim to bring this potential one-time therapy to the clinic in the first half of 2025.”

SGT-1001 is designed to address the underlying genetic cause of Stargardt disease. It consists of a full-length ABCA4 gene construct and mRNA coding for the SaliogaseTM enzyme, SalioGen’s proprietary mammalian-derived bioengineered enzyme. SGT-1001 utilizes a proprietary lipid nanoparticle that is delivered subretinally and will be dosed only once. Preclinical studies show promising gene integration in photoreceptors and retinal pigment epithelium (RPE) cells, as well as sufficient expression of ABCA4 to reduce levels of lipofuscin A2E associated with macular degeneration in a validated animal efficacy model. As measured by Optical Coherence Tomography (OCT), tolerability in non-human primates was consistent with a commercially available gene therapy administered through sub-retinal injection.

SalioGen will present preclinical data supporting SGT-1001 at the May 2024 annual meetings of the Association for Research in Vision and Ophthalmology (ARVO) and the American Society of Gene & Cell Therapy (ASGCT).

About Stargardt Disease

Stargardt disease is an inherited eye disease that causes progressive central vision loss. It is the most common inherited disease of the macula, with an estimated 30,000 people affected in the United States. Mutations in both copies of a person’s ABCA4 gene are the most common cause of Stargardt disease, and more than 1,000 different mutations in ABCA4 can lead to the disease. There are no treatments for Stargardt disease, but genetic medicine has the potential to address the underlying genetic cause of the disease. Although ABCA4 has proven to be a difficult research target given its size (6.8kb), scientists are making important progress for patients.

About SalioGen Therapeutics

SalioGen Therapeutics is developing next-generation genetic medicines for patients using its novel Gene CodingTM technology. Gene Coding is a non-viral method of integrating large or multiple whole genes into the genome at precise locations without double-strand breaks or guide RNA. SalioGen’s lead programs focus on one-time treatments for Stargardt disease and cystic fibrosis. The company is also developing its Gene Coding technology for additional indications, such as other inherited retinal diseases, and to engineer CAR-T cells for cancer and autoimmune conditions. SalioGen is headquartered in Lexington, Massachusetts. For more information, visit SalioGen.com and follow SalioGen on LinkedIn.

Media Contact:

Lori Rosen
LDR Communications
lori@ldrcommunications.com

Investor Relations

Media Relations

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.