Science.

Inspired by each patient and equipped with powerful science, we work together to confront the challenges of treating complex diseases.

Our approach is designed to accelerate the traditional gene therapy development timeline because patients and families are waiting.

Advancing safe and effective gene therapy treatments.

We are targeting genetic diseases with an aim to restore appropriate genetic function to make a positive impact.

Gene therapy involves introduction of healthy copies of genes into the body with the aim of restoring the function of target cells. Our gene therapies are specifically designed to target the root causes of patients’ conditions across a variety of diseases, including: galactosemia; severe neurodevelopmental disorders driven by mutation or deletion in the SHANK3 gene, including austism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS); and Type 1 diabetes. We are journeying beyond rare to fiercely pursue diseases that affect larger numbers of people.

Individuals with severe genetic diseases need therapies that are safe and effective – and they need them as soon as possible. To accomplish this, we leverage our unparalleled expertise and best-in-class manufacturing process intended to minimize development risk and accelerate delivery of safe, effective therapies. Jaguar is harnessing the proven and well-characterized adeno-associated virus (AAV) platform for its initial pipeline programs while continuously evaluating next-generation technologies, including novel capsids and delivery methods for future programs.

AAV-based delivery.

Jaguar’s first three gene therapy programs use AAV-based delivery, meaning AAV functions as a type of vehicle (or vector) used to deliver functioning genes into the target cells. AAV has been shown to be an effective vector because it is nonpathogenic yet incredibly efficient at gaining access to the target cells. To be used as a vector for gene delivery, the viral DNA of AAV is removed and replaced with a gene that is intended to have a therapeutic benefit for a patient suffering from a genetic disease. After the AAV vector delivers its genetic payload to the nucleus of a cell, the gene is then transcribed and translated to produce a functional protein. The gene will exist as an episome and continue to replicate in the nucleus, separately from the chromosomes. The patient’s body then breaks down and processes the AAV vector. AAV-based gene therapy is a proven, well-characterized method that may provide a lifetime of benefit from a one-time administration.

Understanding the rapid pace at which the gene therapy field is evolving, Jaguar is constantly looking to the future and evaluating next-generation gene-delivery technologies.

Designed to prioritize safety and accelerate timelines.

Our team of experts has significant experience harnessing the AAV gene therapy platform to minimize development risk. Ultimately, this enables us to expedite the traditional drug development process, while still ensuring our products are as safe as possible for patients.

Purer products may mean safer, more effective therapies.

Achieving CMC (chemistry, manufacturing and controls) excellence holds the potential to transform the gene therapy field as we know it today; which is why, at Jaguar Gene Therapy, our team is hyper-focused on creating the purest products possible.

With our next-generation purification processes, we can limit product and process-related impurities and ultimately provide the best, most efficacious medicines to patients.

speed-to-clinic speed-to-market

Our commitment from the start:

Product purity and patient safety.

Our team’s experience in developing a scalable and commercially viable gene therapy manufacturing process at the outset of each program has guided our approach to invest early and often in manufacturing. This enables us to adapt and move with agility to overcome manufacturing hurdles by focusing on quality in the preclinical and early manufacturing stages of development.

Owning the capability to manufacture multiple gene therapies from process development to commercial Good Manufacturing Practices (GMP) scale allows us to continuously improve the manufacturing process and provides better control over outcomes. Our Processes Sciences Laboratory in Cary, NC enables our team to produce commercially viable product for our preclinical studies. Meanwhile, our GMP facility in Durham, NC – once fully built-out – is where we will manufacture our commercial-scale product. Our approach of creating scalable and commercially viable constructs right from the start at these facilities is meant to expedite timelines so we can launch and deliver treatments to patients as safely and rapidly as possible.

For more information on our specific programs, please visit our Pipeline.