Our unrelenting commitment to you.

Advocate boldly, care fiercely.

We are fiercely compassionate and fearlessly dedicated to the patients and families we serve.

To help achieve our mission to accelerate breakthroughs in gene therapy, we are fiercely compassionate and fearlessly dedicated to the patients and families we serve. We operate according to our core values of Integrity, Agility and Collaboration, partnering early with patient communities, listening to and understanding the experiences of patients and families affected by severe genetic diseases. We integrate patient and family feedback into the drug development process to guide goals and actions. We respectfully interact with patients and families on their journeys by creating brave spaces for their input and engaging in honest and transparent two-way conversations.

We care deeply and work with urgency, knowing from experience that together, we fight on.

“At Jaguar, patients and families are our north star. You inspire each of us to get out of bed and work hard every day. We listen closely to hear your perspectives. We purposefully include the community as partners in the drug development process. We hope that by working together we can support patients and families on their journeys, and advance new treatment options for those with severe genetic diseases.”

“It is a privilege to stand alongside patients and families in the fight against severe genetic diseases. We are committed to listening, learning and driving forward our programs as quickly and safely as possible. Patients and families inspire us every day, and we promise to do everything we can to translate what’s possible into transformative solutions.”

How we partner.

The Jaguar Patient Advocacy team serves as the primary contact between Jaguar and the patient community. We build our relationships with patients, families and patient communities on a foundation of trust and compassion. Our team is guided by the following principles:

  • We collaborate with families and patient advocacy organizations to identify and advance shared goals.

  • We recognize patients and families as experts. To best support them, we must actively listen to and learn from their unique perspectives and experiences.

  • We rely on the patient community to inform inputs regarding our clinical programs, including clinical trial protocols, procedures and designs.

  • We share transparent and timely updates to keep patient communities apprised of our progress and recognize how their efforts contribute to each success.

  • We comply with all guidelines and regulations that ensure the integrity and safety of clinical research, as well as the appropriate and timely sharing of balanced and accurate information.

Please reach out to us anytime at patientadvocacy@jaguargenetherapy.com.

Resources for patients.

Patient advocacy organizations are independent and trusted resources for patients and families. They can provide reliable information about genetic diseases, research opportunities, support and ways to get involved with the community.



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 uridylyltransferase (GALT) enzyme which causes a toxic buildup of galactose and its metabolites including Gal-lP 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 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.

To learn more about Type 1 galactosemia, view the Understanding Type 1 Galactosemia roundtable discussion, download our information sheet and download the Galactosemia Foundation’s resource Navigating Galactosemia Life Stages: A Handbook for the Galactosemia Community.

Chronic exposure to multiple toxic metabolites can result in patients experiencing lifelong complications.

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. Currently, there is no treatment available for disorders that result from a SHANK3 mutation or deletion. Jaguar is advancing JAG201, an investigational gene therapy currently in preclinical development that aims to deliver appropriate SHANK3 genetic function via the AAV9 vector to treat the root cause of the disease and rescue functional and behavioral abnormalities.

Read our latest Community Letter and FAQ about the JAG201 program.

View select slides from the CureSHANK webinar held on February 22, 2024.

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, approximately 1.5 million individuals are living with Type 1 diabetes. 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.

Our programs.

Jaguar Gene Therapy 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. The company is also working on gene therapies with the potential to help individuals with a genetic cause of autism spectrum disorder, Phelan-McDermid syndrome and/or neurodevelopment disorders with a SHANK3 mutation or deletion, and Type 1 diabetes. Visit our Pipeline to learn more.

These programs are early in the development process. We’ll be sure to share updates and more information as development progresses.


1Galactosemia. National 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