Andelyn Biosciences Forms Partnership with Jaguar for Phelan-McDermid Syndrome Gene Therapy

Advancing Gene Therapy for Phelan-McDermid Syndrome

In recent weeks, Andelyn Biosciences has made headlines with the announcement of its partnership with Jaguar Gene Therapy, a collaboration that aims to significantly advance the treatment options for patients suffering from Phelan-McDermid syndrome (PMS). This groundbreaking effort focuses on the manufacturing of JAG201, an investigational gene therapy that targets SHANK3 haploinsufficiency, a leading genetic cause of autism spectrum disorder.

The Challenge of Phelan-McDermid Syndrome

Phelan-McDermid syndrome affects individuals in profound ways. Those diagnosed with PMS often deal with lifelong repercussions that impact their neurobehavioral, motor, social, and cognitive abilities, resulting in the need for constant supervision and care. Traditional treatment options have neither targeted the underlying disease nor offered effective solutions, as they focus primarily on managing individual symptoms rather than addressing the root cause.

According to the current medical landscape, there are no approved medications specifically designed for PMS or SHANK3-related autism, leaving a significant gap in treatment availability. This makes the collaboration between Andelyn and Jaguar even more critical, as it brings hope to patients and families often left hopeless by conventional options.

Andelyn's Expertise in Gene Therapy Manufacturing

As a leading Contract Development and Manufacturing Organization (CDMO), Andelyn Biosciences specializes in the development of cell and gene therapies. Their deep expertise in adeno-associated virus (AAV) vector manufacturing is essential for the successful execution of JAG201's late-stage Process Performance Qualification (PPQ) manufacturing phase. The company is well-equipped with state-of-the-art facilities in Columbus, Ohio, and a track record that includes over 450 clinical batch productions and collaborations across 75 global clinical trials.

In the realm of gene therapies, effective manufacturing processes are vital to ensure the quality, safety, and efficacy of therapeutics. Andelyn's commitment to upholding Good Manufacturing Practice (GMP) principles ensures that these stringent standards are met every step of the way. Matt Niloff, Andelyn's Chief Commercial Officer, emphasized the shared passion between both companies to expedite safe and effective gene therapies to patients enduring critical health challenges.

What Makes JAG201 Unique

JAG201 aims to provide a new avenue of treatment by delivering a functioning SHANK3 minigene via AAV9 vectors to the neurons in the central nervous system. This gene replacement therapy is carefully engineered to help neurons that lack SHANK3 levels, ultimately restoring synaptic function required for cognitive and communicative development.

The potential impact of JAG201 on the lives of those with Phelan-McDermid syndrome cannot be overstated. By addressing the key genetic deficiencies that contribute to the symptoms of PMS, this therapy could facilitate milestones that are typically absent or significantly delayed in these affected individuals.

Commitment to Patient-Centric Solutions

Rob Mancino, Senior Vice President at Jaguar Gene Therapy, echoed the importance of this partnership, highlighting Jaguar's commitment to excellence in manufacturing processes that prioritize purity and potency. As both organizations continue to work hand-in-hand, they not only advance their respective missions but also contribute immensely to the broader field of gene therapy.

As gene therapy research progresses, partnerships like that of Andelyn and Jaguar Gene Therapy instill hope for many families navigating the challenges of genetic diseases. Their commitment to developing effective solutions reaffirms the importance of innovation in medicine, particularly for rare conditions like Phelan-McDermid syndrome. This collaboration stands as a testament to the potential that exists when focused expertise aligns with a shared purpose—bringing new treatments to patients who need them most.