ametefgene parvec (AB-1005) / Bayer, AskBio - LARVOL DELTA

5-Year Data Support Safety of MRI-Guided AAV2-GDNF Gene Therapy in Advanced Parkinson Disease (NeurologyLive) - "Across the 5-year follow-up period, 562 adverse events (AEs) were recorded, 45 of which were considered possibly or probably related to the study drug. Thirteen serious AEs occurred, none of which were attributed to AAV2-GDNF...There was similarly no significant change in LEDD over the study period (P = .10). Mean LEDD decreased descriptively following treatment and remained lower through approximately 18 months before gradually increasing at later time points. The increase at 60 months was substantially influenced by a marked increase in medication requirements in 1 participant. Investigators also found no significant changes in Hoehn and Yahr staging (P = .09), Lang-Fahn dyskinesia measures, mRS scores (P = .97), or Schwab and England Activities of Daily Living scores (P = .73)."

P1 data • Parkinson's Disease

A Study of AB-1005 Gene Therapy in Japanese Adults With Moderate Parkinson's Disease (J-REGENERATE-PD) (clinicaltrials.gov) - P2 | N=8 | Not yet recruiting | Sponsor: Bayer | Trial completion date: Jun 2032 ➔ Dec 2032 | Initiation date: Jul 2026 ➔ Jan 2027 | Trial primary completion date: Dec 2028 ➔ Jun 2029

Trial completion date • Trial initiation date • Trial primary completion date • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Prescriptive Infusion Algorithm (PIA) (clinicaltrials.gov) - P2 | N=18 | Not yet recruiting | Sponsor: AskBio Inc | Trial completion date: Mar 2028 ➔ Nov 2028 | Initiation date: Jul 2026 ➔ Nov 2026 | Trial primary completion date: Dec 2027 ➔ Apr 2028

Trial completion date • Trial initiation date • Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease

Prescriptive Infusion Algorithm (PIA) (clinicaltrials.gov) - P2 | N=18 | Not yet recruiting | Sponsor: AskBio Inc | N=12 ➔ 18

Enrollment change • CNS Disorders • Movement Disorders • Parkinson's Disease

Explaining early gene therapy research of AB-1005 (GDNF gene therapy) for Parkinson’s disease (PD): patients’/caregivers’ perspective (WPC 2026) - "Efforts to understand the burden of PD on patients/caregivers are crucial for advancing and communicating clinical research and treatment. Results from this study suggest that AB-1005 is well-tolerated and may benefit people with mild to moderate disease by preventing disease progression and reducing existing medication burden. Published in: Van Laar AD, et al."

Clinical • Gene therapy • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease • Sleep Disorder

Evaluation of AAV capsid variant transduction of striato-nigral network using TH-GFP transgenic mice. (ASGCT 2026) - "Conclusion Current work is ongoing to progress the lead capsid to gene therapy efficacy study in PD models and compare efficacy in group studies in other genetic dopamine disorders using AAV2. The identification of an optimal AAV dopaminergic capsid will accelerate the clinical progression of gene therapies for striatal affected neurological disorders."

Preclinical • CNS Disorders • Gene Therapies • Genetic Disorders • Huntington's Disease • Movement Disorders • Parkinson's Disease • Napsin A

AB-1005 Long-Term Follow-up Study (clinicaltrials.gov) - P=N/A | N=132 | Enrolling by invitation | Sponsor: AskBio Inc

Trial initiation date • CNS Disorders • Gene Therapies • Movement Disorders • Multiple System Atrophy • Parkinson's Disease

J-REGENERATE-P: A Study of AB-1005 Gene Therapy in Japanese Adults With Moderate Parkinson's Disease (clinicaltrials.gov) - P2 | N=8 | Not yet recruiting | Sponsor: Bayer

New P2 trial • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

AskBio's Viralgen to Supply Commercial-Scale Drug Product for REGENERATE-PD Investigational Gene Therapy Phase II Trial for Parkinson’s Disease (The Manila Times) - "This enables AskBio to now advance its REGENERATE-PD Phase II clinical trial in the United States with the material produced at the commercial manufacturing facility of AskBio’s wholly owned subsidiary, Viralgen, with other trial sites to follow, using an intensified, high-efficiency, next-generation, large-scale suspension manufacturing process that provides a consistently high-purity product....AskBio is also exploring ametefgene parvec in participants in the United States with the parkinsonian subtype of MSA-P in a fully enrolled Phase I clinical trial to assess the preliminary safety, tolerability, and efficacy for this rapidly progressing neurodegenerative condition."

Commercial • Trial status • Multiple System Atrophy • Parkinson's Disease

ASK-PD5-CS202: A study to improve the delivery of AB-1005 gene therapy into the brain (clinicaltrialsregister.eu) - P1/2 | N=12 | Not yet recruiting | Sponsor: Askbio Inc., AskBio France

New P1/2 trial • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

AskBio’s AB-1005 and AB-1002 Receive Pioneering Regenerative Medical Product Designation in Japan (The Manila Times)

Japanese regulatory • Congestive Heart Failure • Parkinson's Disease

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD) (clinicaltrials.gov) - P2 | N=127 | Recruiting | Sponsor: AskBio Inc | N=87 ➔ 127 | Trial completion date: Nov 2027 ➔ Aug 2028 | Trial primary completion date: Nov 2027 ➔ Aug 2028

Enrollment change • Trial completion date • Trial primary completion date • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Clinical Progress and Plans for Aav2GDNF Gene Therapy for Parkinson's Disease (ESGCT 2024) - No abstract available

Clinical • Gene therapy • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

AskBio Inc…announced that the first European participants have been randomized in REGENERATE-PD, a Phase 2 clinical trial in participants with moderate-stage Parkinson’s disease (PD). (GlobeNewswire) - "REGENERATE-PD is estimated to enroll approximately 87 participants across clinical centers in Germany, Poland, the United Kingdom, and the United States."

Trial status • Parkinson's Disease

Phase II gene therapy trial REGENERATE-PD with AB-1005 initiated randomizing participants in United Kingdom and Poland, with Germany following shortly and ongoing in the USA (Bayer Press Release)

Trial status • Parkinson's Disease

AskBio Announces Completion of Enrollment in Phase 1 Clinical Trial of AB-1005 Gene Therapy for Multiple System Atrophy-Parkinsonian Type (MSA-P) (GlobeNewswire)

Enrollment closed • Multiple System Atrophy

Bilateral Putaminal Convection of Adeno-Associated Virus Vector Serotype 2 Glial Cell Line-Derived Neurotrophic Factor Gene Therapy in Parkinson's Disease May Provide Glial Cell Line-Derived Neurotrophic Factor Transgene Expression to Caudate Nucleus: Potential Role of Perivascular and Other Low Resistance Leakage Pathways. (PubMed, Neurosurgery) - "These findings highlight anatomical and perivascular interconnections as potential contributors to off-target dispersion. Although no clinical benefit was observed, this study underscores the potential for targeted caudate transduction with optimized infusion strategies. The results emphasize the need for further exploration of perivascular dynamics and volumetric coverage in future gene therapy trials for Parkinson disease and other central nervous system disorders."

Journal • Preclinical • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

GDNF Gene Therapy for Multiple System Atrophy (clinicaltrials.gov) - P1 | N=9 | Active, not recruiting | Sponsor: Brain Neurotherapy Bio, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2028 ➔ Aug 2028 | Trial primary completion date: Dec 2025 ➔ Aug 2026

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Gene Therapies • Movement Disorders • Multiple System Atrophy

AskBio Announces Publication of Complete Results of Phase 1b Trial of AB-1005 Gene Therapy in Participants with Parkinson’s Disease in Movement Disorders (GlobeNewswire) - P1b | N=11 | NCT04167540 | Sponsor: Brain Neurotherapy Bio, Inc. | "AskBio Inc...announced today the recent publication of the complete results of its Phase 1b trial of AB-1005, a glial cell line-derived neurotrophic factor (GDNF) investigational gene therapy for the treatment of Parkinson’s disease (PD), in Movement Disorders, an official peer-reviewed journal of the International Parkinson and Movement Disorder Society....The publication, which is available online, states that treatment with AB-1005 was well tolerated with no serious adverse events related to GDNF gene therapy in all 11 participants and is associated with numerical stability in clinical assessment readouts (mild cohort) and improvement (moderate cohort) in clinical assessments of motor function at 18 months post-gene transfer."

P1 data • Movement Disorders • Parkinson's Disease

Intraputaminal Delivery of Adeno-Associated Virus Serotype 2-Glial Cell Line-Derived Neurotrophic Factor in Mild or Moderate Parkinson's Disease. (PubMed, Mov Disord) - "Bilateral intraputaminal AAV2-GDNF gene therapy was well tolerated and associated with numerical stability (mild cohort) and improvement (moderate cohort) in clinical assessments at 18 months posttreatment."

Journal • Preclinical • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

AskBio Receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease investigational gene therapy (Bayer Press Release) - "AskBio...announced that investigational gene therapy AB-1005 for the treatment of Parkinson’s disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA)....The FDA determined that AB-1005, an investigational gene therapy intended to slow disease progression and improve motor outcomes in patients with PD, met the criteria for RMAT designation. This decision follows a review of information and data provided by AskBio, including clinical evidence from the open label, uncontrolled study Phase Ib trial of AB-1005. AskBio’s 36-month Phase Ib data showed that the administration of AB-1005 was well tolerated with no product-related serious adverse events."

FDA event • Gene Therapies • Parkinson's Disease

First participants randomized in AskBio Phase II gene therapy trial for Parkinson’s disease (Bayer Press Release) - "AskBio...announced that the first participants have been randomized in its Phase II clinical trial in patients with Parkinson’s disease (PD)....The objective of this randomized, double-blind, Phase II clinical trial is to evaluate the safety and efficacy of AB-1005 delivered to the putamen in adult participants aged 45–75 years with moderate-stage PD. REGENERATE-PD is estimated to enroll approximately 87 participants across clinical centers that are being opened in the United States, Germany, Poland, and the United Kingdom."

Trial status • Parkinson's Disease

AskBio…presented 36-month Phase Ib data at the International Congress of Parkinson’s Disease and Movement Disorders (Bayer Press Release) - P1b | N=11 | NCT04167540 | Sponsor: Brain Neurotherapy Bio, Inc. | "The data demonstrated that administration of AB-1005 was well tolerated with no attributed serious adverse events. The Moderate PD cohort showed trends for improvement or stability on several motor scales at 36 months, including Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and motor diaries, and trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]). Most participants in the Mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD."

P1 data • Parkinson's Disease

Preliminary Efficacy of Bilateral Intraputaminal Delivery of GDNF Gene Therapy (AAV2-GDNF; AB-1005) in Parkinson’s Disease: 18-Month Follow-Up From a Phase 1b Study (MDS Congress 2024) - "Patients with mild or moderate PD continued to show stability and/or improvement in PD-specific clinical rating scales at 18 months after AAV2-GDNF (AB-1005) delivery. Data from longer-term follow-up will be available and presented Table."

Clinical • Gene therapy • P1 data • CNS Disorders • Parkinson's Disease

REGENERATE-PD: A Phase 2, Randomized, Double-Blind, Surgery-Controlled Study of GDNF Gene Therapy (AAV2-GDNF; AB-1005) for Moderate Stage Parkinson's Disease (MDS Congress 2024) - P2 | "The trial is expected to begin enrollment by mid-2024 (NCT06285643). Study schema"

Clinical • Gene therapy • P2 data • Surgery • CNS Disorders • Parkinson's Disease