Our unrelenting commitment to you.
Advocate boldly, care fiercely.
“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.”
“We strive to become part of the community that will help make advancements across the board, not just in treatment, but in awareness, diagnosis and accurate disease understanding.”
“We strive to understand the needs of the community and amplify the patient voice. We partner with the community early and often to ensure that we can meaningfully support patients and their families, and to ensure their concerns are being heard throughout the drug development process.”
“We know those dealing with severe genetic diseases often have limited treatment options. We have a deep bench of proven experts in this area, and we believe it is our responsibility to apply that extensive experience to help address these unmet needs.”
“The patient community is a core part of our team, and they are the reason why we come to work every day. We are committed to listening to their experiences and making sure their voices are heard every step of the way. They are experts, and we are humbled to work with them.”
“We strive to build collaborative, transparent and inclusive two-way relationships with patient advocacy groups, and make every effort to understand and solidify the science while working alongside the patient community.”
“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.”
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:
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.
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.
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.
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