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As autism treatment and support continue, one significant development emerges from Lake Forest’s biotechnology firm, Jaguar Gene Therapy. The company has recently gained federal approval to initiate a novel therapeutic intervention that targets a genetic subtype of autism.

In other words, as disclosed in yesterday’s announcement, they have received the U.S. Food & Drug Administration’s green light to commence a Phase 1 clinical trial.

Upcoming trial

The upcoming trial is slated to begin with the treatment of the first pediatric patient using the innovative JAG201 therapy, anticipated in the first quarter of the forthcoming year. Following the evaluation within the pediatric cohort, the trial’s scope will extend to encompass adult participants, thereby advancing the understanding and potential application of this groundbreaking treatment across different age groups.

The target population for this investigational therapy includes individuals with Autism related conditions rooted in genetic anomalies, specifically addressing complications arising from the Shank3 gene deficiency, also recognized as haploinsufficiency. This genetic condition, formally identified as Phelan-McDermid syndrome, presents a significant challenge, given its profound impact on neurological development and function. 

Early intervention within the developmental trajectory

According to Dr. Alexander Kolevzon, an esteemed professor, the imperative lies in initiating early intervention within the developmental trajectory of affected patients.

Targeting the foundational impairments instigated by the Shank3 deficiency during the critical periods of growth and development, there exists a substantially enhanced prospect for therapeutic efficacy.  The approach aims to rectify functional deficits through the administration of a single, precisely delivered intracerebroventricular injection. In layman’s terms, this means that the therapy administers a corrective dose directly into the brain’s ventricular system, where it is then distributed throughout the neural architecture.

Then, it propagates its effect across the comprehensive expanse of the central nervous, including the brain and spinal cord. If successful, this innovative therapy aims to rectify the insufficient expression of the Shank3 gene, which is critical for maintaining optimal synaptic functions integral to cognitive processes.

The remediation of these synaptic pathways is anticipated to facilitate significant improvements in neural communication, thereby mitigating the symptoms associated with Shank3 haploinsufficiency.  

There exists no Shank3 approved remedy

As of now, no approved therapeutic interventions specifically targeting Shank3 haploinsufficiency exist. The innovative strides made by Jaguar Gene Therapy resonate deeply within the autism community, bringing hope for a future where genetic conditions may be effectively managed. 

Autism Support in Chicago, IL

For families and individuals navigating autism, Illinois Autism Center is here to offer unwavering support, awareness, and advocacy.

Our organization is steadfast in its commitment to providing resources, education, and compassionate treatment to those affected by autism.