On this World Parkinson's Day 2025, a stark reality emerges: nearly 1 million individuals in the U.S. and over 6 million worldwide grapple with Parkinson's disease (PD). The symptoms vary tremendously from person to person, and while there are treatment options available, a definitive cure remains elusive, underscoring a critical need within the patient community.

One pivotal area of research is the advancement of cell and gene therapies. Numerous companies and academic institutions are diving headfirst into developing innovative treatments, with several significant clinical trials underway. As we honor this day each year on April 11, CGTLive® reflects on the impressive strides made in these therapies over the past year.

Groundbreaking RMAT Designation for AskBio's Gene Therapy

Just announced: AskBio's experimental gene therapy AB-1005, designed to combat PD, has earned the coveted Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. This revolutionary therapy utilizes AAV2 (Adeno-Associated Virus) technology and showcases promising results from a Phase 1b trial, which is now transitioning to a sham-controlled Phase 2 trial across multiple countries.

CEO Gustavo Pesquin expressed enthusiasm: "This milestone could accelerate the development of our important investigational therapy, underscoring its potential benefits for patients and the medical community. We’re eager to collaborate with the FDA to advance our program."

Capsida Biotherapeutics Makes Waves with CAP-003

In another exciting development, Capsida Therapeutics revealed that its gene therapy, CAP-003, effectively modifies the disease in preclinical research. Administered intravenously, this therapy showed remarkable results in nonhuman primates, significantly outpacing previous treatments and leading to heightened GCase protein activity. CEO Peter Anastasiou stated, "This novel gene therapy could normalize GCase activity with just a single infusion, offering the potential for substantial disease modification."

TED-A9 Therapy: A Ray of Hope for PD Symptoms

July 2024 witnessed promising developments from the TED-A9 cell therapy, which demonstrated safety and efficacy in a small Phase 1/2 clinical trial in South Korea. Doctors reported that early participants showed signs of symptom improvement without adverse effects related to transplant surgery. As Professor Dong-Wook Kim noted, TED-A9 aims to replace lost dopaminergic neurons, potentially transforming treatment protocols for PD.

BlueRock's Cell Therapy Gains FDA Recognition

Adding to the excitement, BlueRock Therapeutics' pioneering cell therapy bemdaneprocel received RMAT designation from the FDA as well. This therapy seeks to restore functionality to neurons compromised by Parkinson's, demonstrating substantial promise in their Phase I clinical trials. President Seth Ettenberg remarked, "We are optimistic about the potential benefits this therapy could bring to PD patients, eager to embark on Phase II studies soon!"

NKGen's Bold Steps into Clinical Trials

Held with high anticipation, NKGen's autologous NK cell therapy, SNK01, has received FDA clearance for its clinical trials, targeting neurodegenerative diseases like PD. This exciting advancement represents a significant conversational shift towards tackling both the cellular changes and inflammation that underpin neurological decline. CEO Paul Y. Song articulated the importance of this progress, highlighting the urgent need for effective treatments.

As World Parkinson's Day 2025 unfolds, these advancements in cell and gene therapy shine a hopeful light on the future of Parkinson's treatment, offering the potential for more effective interventions and improved quality of life for millions.