Accelerating gene therapies for patients.

At Jaguar Gene Therapy, we are laser-focused on our mission of accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases.

The Jaguar Gene Therapy pipeline.

We are advancing our initial pipeline that targets diseases with significant unmet need in sizeable patient populations. We aim to make a big impact because people’s lives depend on the work we do. We are journeying beyond rare to fiercely pursue diseases that affect larger numbers of people. 

Through our strategic partnerships, we have access to key academic institutions, renowned academic experts, world-class laboratories and essential suppliers. We leverage these, as well as our own deep connections within the industry, to thoughtfully continue building a diversified pipeline of gene therapy treatments, technologies and approaches.

Target Identification

Discovery

Preclinical

Clinical

JAG101

Jaguar is advancing JAG101, an investigational gene therapy currently in preclinical development that aims to deliver a gene replacement solution to address the root cause of Type 1 galactosemia by delivering the functional GALT gene via the AAV9 vector. Gene therapy offers the opportunity to have immediate impact to reduce multiple toxic metabolites simultaneously, notably galactose, Gal-1P and galactitol, bringing them closer to a normal level to positive change the clinical course of the disease and mitigate longer-term complications. Jaguar has research agreements in place for the program with Emory University and the University of Utah, which produced encouraging preclinical proof-of-concept data in animals.

Type 1 Galactosemia

Type 1 galactosemia is 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 and 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 in functional galactose-1-phosphate uridylytransferase (GALT) enzyme, which causes a toxic buildup of galactose and its 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

References:

1National Organization for Rare Disorders (NORD) Rare Disease Database. 2019. Accessed October 27, 2021. https://rarediseases.org/rare-diseases/galactosemia/

2Berry GT. Classic galactosemia and clinical variant galactosemia. February 4, 2000. Updated March 11, 2021. Accessed October 27, 2021. https://www.ncbi.nlm.nih.gov/books/NBK1518/

3Rubio-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. https://doi.org/10.1093/humupd/dmp038

4GALT gene. MedlinePlus. August 18, 2020. Accessed October 27, 2021. https://medlineplus.gov/genetics/gene/galt/

5Genetic and Rare Diseases (GARD) Information Center. 2021. Accessed October 27, 2021. https://rarediseases.info.nih.gov/diseases/2424/galactosemia

JAG101

Type 1 Galactosemia

Preclinical

JAG201

Jaguar is advancing JAG201, an investigational gene therapy currently in preclinical development that is intended to deliver appropriate SHANK3 genetic function via the AAV9 vector to treat the root cause of the disease and rescue functional and behavioral abnormalities. The program is exclusively licensed from Broad Institute of MIT and Harvard, where encouraging preclinical proof-of-concept animal data was produced.

A Genetic Cause of Autism Spectrum Disorder, Phelan-McDermid Syndrome and Other Severe Neurodevelopmental Disorders with SHANK3 Mutation or Deletion

Individuals can be diagnosed with autism spectrum disorder (ASD), Phelan-McDermid syndrome and/or other severe neurodevelopmental disorders if a mutation or deletion in the SHANK3 gene is present. Disorders that result from a SHANK3 mutation or deletion are associated with prefrontal lobe dysfunction, autism, moderate to severe intellectual disability, absent or severely delayed speech, and neurodevelopmental behavioral abnormalities among other characteristics. SHANK3 is a leading candidate gene thought to cause ASD. In the United States, approximately 30,000 individuals living with ASD have a SHANK3 mutation or deletion. [2] Currently, there is no treatment available for disorders that result from a SHANK3 mutation or deletion.

JAG201

Disorders with SHANK3 Mutation or Deletion

Preclinical

JAG301

Jaguar Gene Therapy is advancing JAG301, an investigational AAV-based gene therapy currently in preclinical development that works 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. This program is exclusively licensed from Tulane University, where encouraging preclinical proof-of-concept data was produced. Additionally, Jaguar has a research agreement in place with Tulane.

Type 1 Diabetes

Type 1 diabetes is an autoimmune disease that destroys beta cells, which are specialized cells in the pancreas that produce insulin. A lack of insulin results in the inability of cells to use glucose for energy and unregulated glucose levels in the blood, which can lead to long-term complications, including frequent hospitalizations, blindness, heart disease, stroke, kidney disease and nerve damage. No cure is available today, and treatment is based on diet, insulin injections and continuous glucose monitoring. In the United States, there are approximately 1.5 million individuals living with Type 1 diabetes.

JAG301

Type 1 Diabetes

Preclinical