World Parkinson's Day 2025: Looking Back at Progress for Cell and Gene Therapy
In observance of World Parkinson's Day, held annually on April 11, we took a look back at the past year's news in cell and gene therapy for PD.
According to the Michael J. Fox Foundation, almost 1 million people in United States live with Parkinson disease (PD) and more than 6 million people in the world live with PD. Notably, the symptoms of PD and their severity vary widely, and can be different for every person. Although some treatment options for the disease exist, there is currently no cure and great unmet need remains for the patient community.
An important area of interest for new therapeutic development in PD is cell and gene therapy. A number of companies and academic institutions are now pursuing the development of such advanced treatments, with several clinical trials currently active. In honor of World Parkinson's Day, observed annually on April 11 by the patient and clinician communities, CGTLive® is taking a look back at the progress that has been made in cell and gene therapy for PD over the past year. Click the "READ MORE" buttons for more details and information about each item.
AskBio Garners RMAT Designation for Parkinson Disease Gene Therapy AB-1005
February 24, 2025 — Bayer subsidiary Asklepios BioPharmaceutical (AskBio)'s AB-1005 (AAV2-GDNF), an investigational AAV vector serotype 2 (AAV2)-based gene therapy intended to treat PD, has received regenerative medicine advanced therapy (RMAT) designation from the FDA.
The FDA’s decision was based on a review of findings from a phase 1b clinical trial (NCT04167540), which has passed its primary completion date but is continuing to collect long-term follow-up data. Notably, AB-1005 is also now being evaluated for PD in a sham-controlled phase 2 clinical trial (REGENERATE-PD; NCT06285643), which recently began the process of randomly assigning enrolled participants in the United States in January 2025. AskBio anticipates that REGENERATE-PD will open additional sites for enrollment in the US, Germany, Poland, and the United Kingdom before the midway point of 2025.
“The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with PD and their loved ones,” Gustavo Pesquin, MBA, the chief executive officer of AskBio, said in a statement. “This milestone could potentially expedite the development of our important investigational gene therapy program, and it highlights our promising data and the potential of AB-1005 for patients and the medical community. We look forward to working closely with the FDA to accelerate our program.”
Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research
November 5, 2024 — Capsida Therapeutics' CAP-003, an intravenously (IV) delivered gene therapy intended to treat PD associated with GBA mutations (PD-GBA), produced a brainwide RNA expression in nonhuman primates (NHPs) that was more than 200-fold greater than IV administered AAV9 along with substantial increases in GCase protein and enzyme activity compared with untreated NHPs. The data were presented in a late-breaking poster presented at the Society for Neuroscience annual meeting, held October 5-9, in Chicago, Illinois.
"Capsida's wholly owned novel gene therapy offers the potential to normalize GCase activity in patients with a single IV infusion safely, enabling the potential for long-term disease modification and substantial slowing of disease progression," Peter Anastasiou, MBA, the chief executive officer of Capsida, said in statement. "These data give us confidence that we are on track to enter the clinic with CAP-003 in the first half of 2025."
CAP-003 is intended to enable production of the GCase enzyme, the gene for which is mutated in PD-GBA, with the aim of slowing disease progression via restoration of normal enzyme activity. The findings in the NHPs indicated that all CAP-003 doses exceeded the 30% efficacy threshold that would be expected to be required for normalizing GCase activity in human patients. Capsida noted that GCase activity in the NHPs, including within the substantial nigra, was 2-8-fold higher than that threshold.
Dopaminergic Progenitor Cell Therapy Shows Some Symptom Improvement in Parkinson Disease
July 16, 2024 — TED-A9 cell therapy has demonstrated safety with some preclinical efficacy in the first 4 participants with PD treated in a phase 1/2 clinical trial conducted at Severance Hospital of Yonsei University in South Korea led by Professor Jin-Woo Chang, a neurosurgeon and Professor Phil Hyu Lee, a neurologist.
“Although this clinical evaluation only targets the first 3 low-dose patients, but not all 12 subjects, no adverse issues related to transplant surgery or cell safety were observed in one-year post transplantation. Importantly, the clinical results demonstrated very promising efficacy,” Prof. Dong-Wook Kim, Yonsei University College of Medicine and CTO, S.BIOMEDICS, said in a statement. “The results are believed to align closely with the findings from our preclinical in vitro and in vivo studies. We are excited that TED-A9 could be a fundamental treatment that directly replaces dopaminergic neurons lost in patients with PD.”
TED-A9 is a hESC (human embryonic stem cell)-derived midbrain dopaminergic progenitor cell therapy surgically transplanted to the anterior, middle, and posterior sections of the putamen, with 3 tracks per each putamen. The transplanted cells are then expected to mature into dopaminergic neurons and restore dopamine production and motor function.
FDA Recognizes BlueRock’s Parkinson Disease Cell Therapy With RMAT Designation
June 17, 2024 — The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BlueRock Therapeutics’s bemdaneprocel cell therapy for the potential treatment of PD.
“We are excited about the positive data from the bemdaneprocel phase I clinical trial and believe it has great potential to help patients living with PD regain functions they have lost to the disease,” Seth Ettenberg, President and CEO, BlueRock Therapeutics, said in a statement. “Now with this RMAT designation in hand, we look forward to closely collaborating with the FDA to ready this program for phase II clinical studies.”
Bemdaneprocel is a dopaminergic neuron precursor cell therapy and is being evaluated in a phase 1/2 clinical trial (NCT04802733). The trial is primarily assessing the incidence and severity of adverse events (AEs) after administration of the cell therapy. Secondary outcome measures being assessed include cell survival, as measured by change in 18F-DOPA uptake using positron emission tomography (PET); changes in motor function, as measured by MDS-Unified Parkinson's Disease Rating Scale (UPDRS) motor sub-score in the "off" state; changes in waking hours in "Off" state; and long-term safety and tolerability.
NKGen’s NK Cell Therapy SNK01 Cleared for US Clinical Trial in Parkinson Disease
April 30, 2024 — NKGen’s autologous natural killer (NK) cell therapy SNK01 has received clearance of an investigational new drug (IND) application from the FDA for a phase 1/2a clinical trial in patients with PD.
The planned trial will seek to enroll up to 30 patients, 20 of whom will be treated with SNK01, and 10 of whom will receive a placebo. NKGen anticipates that it will dose the first patient in the study in the second half of this year.
“The IND clearance marks a significant milestone as we advance our pipeline of NK cell therapy in neurodegenerative diseases,” Paul Y. Song, MD, the chairman and chief executive officer of NKGen, said in a statement. “This is our second IND approval for SNK01 within the past several months and underscores our focused dedication towards developing safe and effective treatments that target both protein deposition and neuroinflammation for patients with neurodegenerative diseases such as Alzheimer disease and PD. We are excited to start our first clinical trial in PD as there currently is a high unmet medical need in this indication.”
