Cystic Fibrosis

Cystic Fibrosis

Cystic Fibrosis (CF) is a rare genetic disease usually diagnosed at birth via newborn screening. While the severity of CF may vary from person to person, it is a chronic and life-threatening condition that progressively damages the lungs, digestive system, and other organs in the body. According to the Cystic Fibrosis Foundation, approximately 105,000 people around the world live with CF.

Gene

CFTR

Disease-causing Mutations

~2,000

Cause

Mutations in a gene called CFTR cause CF. To have the disease, a person must inherit two copies of the defective gene – one from each parent. There are approximately 2,000 different mutations in CFTR that can cause CF.

In CF, the defective CFTR gene means the CFTR protein doesn’t work correctly. This impairs the body’s ability to move salt and water in and out of cells, producing thick, sticky mucus. Over time, this mucus builds up in the airways and digestive system, making breathing and digesting foods difficult and leading to chronic infections, malnutrition, and other dangerous complications.

Treatment

Traditionally, treating CF involves using multiple therapies daily to maintain lung function, reduce complications, and improve quality of life. More recently, CFTR modulators have been approved specifically to treat CF, but they are not cures. People must take these medicines every 12 hours, and they only work for people who have specific mutations. Currently, about 10% of people with CF are not eligible for these chronic therapies.

At SalioGen, we’re focused on developing a non-viral genetic medicine that integrates a fully functional copy of the CFTR gene into cells in the lungs. Our potential one-time therapy would be the first permanent solution for all people living with CF, regardless of mutation. We work urgently to optimize our Gene Coding™ technology to meet this challenge.

Helpful Resources

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

Cystic Fibrosis Foundation

View resource

Emily’s Entourage

View resource

The National Organization of African Americans with Cystic Fibrosis

View resource

An Extended Familia for Latinos Living with CF

View resource

United States Adult Cystic Fibrosis Association

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.