Samiah Al-Zaidy, MD, on Gene Therapy for Infantile GM1 Gangliosidosis

“There’s a tremendous unmet need [in infantile GM1 gangliosidosis]. Currently, the only option for these families is supportive care. Supportive care consists of offering support to patients and their families with the aim of preventing serious complications and providing comfort care. There is no reversal of the loss of function with supportive care.”

For patients with infantile GM1 gangliosidosis, supportive care is the only treatment option, as no disease-modifying therapies are currently available. As a result, these patients experience rapid disease progression, and typically do not live beyond the first few years of life. Although clinical care is limited by this lack of options, efforts in clinical development are underway.

PBGM01, an investigational adeno-associated virus (AAV) vector-based gene therapy currently being evaluated in the phase 1/2 Imagine-01 clinical trial (NCT04713475), is intended to address this immense unmet need. Passage Bio, the developer of PBGM01, presented updated interim safety and efficacy data from Imagine-01 at the WORLDSymposium 2023, held February 22-26, in Orlando, Florida.

In an interview with CGTLive, Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the promising results seen thus far, pointing to positive signs of efficacy. She noted increases seen with the trial’s high-dose group in activity of beta-galactosidase (β-Gal), the enzyme deficient in patients with GM1 gangliosidosis, and stabilization seen in MRI severity scores for the treated patients. Al-Zaidy also discussed the encouraging safety profile of PBGM01 and future plans for further research with higher doses of the gene therapy.