Jaguar Gene Therapy Announces Samsung and JDRF T1D Fund as New Investors

  • Company’s top-tier biotech investor base expands to include new strategic backers with an aligned vision of the significant impact gene therapy can make for patients and families suffering from devastating conditions
  • Funding proceeds will be used to support ongoing investments in the company’s first-in-class Chemistry, Manufacturing and Controls (CMC) capabilities and advance IND-enabling activities for its initial preclinical programs

LAKE FOREST, Ill.--(BUSINESS WIRE)-- Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations, today announced Samsung Ventures, an investment corporation established to promote the development of new technologies, and the JDRF T1D Fund, a venture philanthropy fund devoted to finding and funding the best cure-oriented therapies for Type 1 diabetes (T1D), have invested in the company.

“We are excited to add such visionary strategic investors to our existing top-tier biotech investor base,” said Joe Nolan, Chief Executive Officer of Jaguar Gene Therapy. “The investments from Samsung and the JDRF T1D Fund will enable further investment in our first-in-class CMC capabilities and help us advance IND-enabling activities for our exciting preclinical pipeline.”

Jaguar will use the proceeds of the new investments, together with its existing cash resources, to continue to expand its CMC capabilities and advance its preclinical pipeline of gene therapy programs targeting: Type 1 galactosemia; a genetic cause of autism spectrum disorder (ASD) and Phelan-McDermid syndrome and other severe neurodevelopmental disorders with a SHANK3 mutation or deletion; and T1D.

“We are impressed by Jaguar’s CMC experience and uncompromising commitment towards the development of the industry leading process development and commercially scalable processes for the manufacture of AAV therapeutics of unparalleled purity, safety and yield. As a global leader in innovation and technology, Samsung appreciates the significance of Jaguar’s mission to accelerate breakthroughs in gene therapies for severe genetic diseases,” said Il Seok Yoon, Chief Investment Officer of Samsung Ventures. “We believe that by investing in companies such as Jaguar, we ultimately enable the continued expansion of AAV gene therapies to help improve the lives of people in need across the globe.”

“As a Fund focused on investing in innovative disease-modifying therapies for T1D, we are thrilled to support the experienced team at Jaguar and their highly promising JAG301 program,” said Katie Ellias, Managing Director, JDRF T1D Fund. “The company’s gene therapy approach has the potential to be a functional cure for T1D by restoring endogenous insulin production in patients as a result of a one-time treatment. The Jaguar team’s proven track record, having overseen the development and launch of one of the first FDA-approved gene therapies, makes them expertly positioned to bring this cure-oriented therapy to T1D.”

Jaguar’s Preclinical Pipeline Programs

Jaguar is advancing three investigational AAV-based gene therapies, all of which are currently in preclinical development.

Jaguar is developing JAG101 for Type 1 galactosemia, a rare genetic disease that can be life-threatening for newborns and cause severe lifelong complications starting as early as the first year of life.1,2,3 Galactosemia affects the body’s ability to make the enzyme that breaks down galactose, a simple sugar the body endogenously produces that is also found in dairy and other foods, including breast milk.1,4,5 Type 1 galactosemia is caused by mutations in the GALT gene, which lead to a severe deficiency of the galactose-1-phosphate uridylyltransferase (GALT) enzyme, which causes a toxic buildup of galactose and other metabolites including Gal-1p and galactitol. This buildup of toxic metabolites is a life-threatening medical emergency in newborns and can contribute to lifelong cognitive, neurological, and speech complications, as well as primary ovarian insufficiency in girls and women.1,2,4,5 Because of its severity, galactosemia is part of newborn screening in all 50 states of the United States and in several other countries.1,2,5 No treatments are currently approved for galactosemia, and there is significant unmet medical need. The current standard of care – a galactose-restricted diet – is insufficient because the body endogenously produces galactose, causing patients to experience chronic complications.2

For the treatment of a genetic cause of ASD and Phelan-McDermid syndrome and other severe neurodevelopmental disorders with a mutation or deletion in the SHANK3 gene, Jaguar is developing JAG201. Currently, no treatment is available for disorders that result from a SHANK3 mutation or deletion. JAG201 aims to deliver appropriate SHANK3 genetic function via the AAV9 vector to treat the root cause of the disease and rescue behavioral abnormalities​.

For the treatment of Type 1 diabetes, an autoimmune disease in which beta cells, the specialized cells in the pancreas that produce insulin, are destroyed, Jaguar is developing JAG301. No cure is available for Type 1 diabetes, and treatment is based on achieving glucose control through diet, insulin injections and glucose monitoring. JAG301 is designed to address the root cause of the disease by producing functional beta cells using the PAX4 gene to convert alpha cells to beta cells, a process called transdifferentiation.

About Samsung Ventures

Samsung Venture Investment Corporation was established to promote promising small and medium-sized companies engaging in the development of new technologies. Our collaboration is based on managerial know-how and trust, and actively investing in future-oriented businesses based on new and innovative technologies that are expected to serve as new growth engines. Please visit our website for more detail;

About JDRF T1D Fund

The JDRF T1D Fund ( is a venture philanthropy fund accelerating life-changing solutions to cure, prevent and treat type 1 diabetes (T1D) through catalytic equity investments. Through its investments in partnership with private capital, including venture capital, corporations and foundations, the T1D Fund seeks to attract the private investment necessary to advance therapeutics, devices, diagnostics and vaccines into the hands of those living with T1D. The T1D Fund invests in areas strategically aligned with JDRF, the leading global organization funding T1D research, with an exclusive focus on supporting the best commercial opportunities. The T1D Fund reinvests any realized gains into new investments to further its mission.

About Jaguar Gene Therapy

Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations. The company is made up of a proven team of experts with unparalleled CMC (Chemistry, Manufacturing and Controls), regulatory, clinical and commercial acumen who have first-hand experience in bringing novel gene therapy treatments to patients and their families. Committed to patient safety and product purity, Jaguar is rapidly advancing an initial pipeline of three programs targeting: 1) Type 1 galactosemia; 2) a genetic cause of autism spectrum disorder and Phelan-McDermid syndrome and other severe neurodevelopmental disorders with a SHANK3 mutation or deletion; and 3) Type 1 diabetes. The company continues to evaluate opportunities to expand its pipeline of investigational gene therapies. For more information, please visit and follow Jaguar Gene Therapy on LinkedIn.


1 Galactosemia. National Organization for Rare Disorders (NORD) Rare Disease Database. 2019. Accessed October 27, 2021.

2 Berry GT. Classic galactosemia and clinical variant galactosemia. February 4, 2000. Updated March 11, 2021. Accessed October 27, 2021.

3 Rubio-Gozalbo ME, Gubbels CS, Bakker JA, Menheere PPCA, Wodzig WKWH, Land JA. Gonadal function in male and female patients with classic galactosemia. Hum Reprod Update. 2010;16(2):177-188.

4 GALT gene. MedlinePlus. August 18, 2020. Accessed October 27, 2021.

5 Galactosemia. Genetic and Rare Diseases (GARD) Information Center. 2021. Accessed October 27, 2021.

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Julie Normart

Kate Neer

Source: Jaguar Gene Therapy

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