Jaguar Gene Therapy Secures FDA IND Approval to Explore JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome
Thursday, February 01, 2024
Jaguar Gene Therapy, a biotechnology firm specializing in advancing gene therapy for severe genetic diseases, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) Application for JAG201. This gene therapy targets a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS), conditions affecting around 30,000 individuals in the United States with SHANK3 mutations or deletions.
With no effective treatments currently available, JAG201 aims to address the root cause of these disorders by delivering functional SHANK3 using the AAV9 vector. Positive preclinical studies in rodents and non-human primates have shown improvements in neurobehavioral, cognitive, and motor function abnormalities with functional SHANK3 delivery.
Jaguar Gene Therapy plans to initiate a Phase I trial for JAG201 in adults with ASD or PMS having SHANK3 mutations or deletions in the second half of the year.
CEO Joe Nolan expressed enthusiasm, stating, "This is a significant milestone for our company but more importantly for the approximately 30,000 individuals living with a genetic form of autism or Phelan-McDermid syndrome."
Clinician Alexander Kolevzon emphasized the need for better treatment options, saying, "My patients and all those with SHANK3 haploinsufficiency deserve a treatment option that addresses the underlying biology."
SHANK3 haploinsufficiency disrupts synaptic function, leading to communication issues between nerve cells. JAG201 delivers a functional SHANK3 minigene through an adeno-associated virus serotype 9 (AAV9) vector to target neurons in the central nervous system. Administered via a one-time unilateral intracerebroventricular (ICV) injection, the therapy aims to transduce haploinsufficient neurons, restore proper SHANK3 levels, and enhance synaptic function crucial for learning and memory.
The program is exclusively licensed from the Broad Institute of MIT and Harvard, based on the work of Dr. Guoping Feng. Feng expressed excitement about the clinical progression of JAG201, citing promising preclinical data.
Phelan-McDermid syndrome (22q13.3 deletion syndrome) arises from SHANK3 haploinsufficiency, affecting around 1 in 10,000 individuals. SHANK3 mutations are also present in approximately 1% of ASD patients, estimated at around 30,000 in the U.S.
A recent Externally Led Patient Focused Drug Development (EL-PFDD) Meeting highlighted the severe impact of PMS on quality of life, the significant unmet medical need, and the inadequacy of existing therapies.
Patient advocacy groups, including CureSHANK and the Phelan-McDermid Syndrome Foundation, expressed excitement about JAG201's clearance for clinical trials, emphasizing the urgent need for effective treatments to address the root cause of these debilitating disorders.
Source: businesswire.com
