MedPath

AB-1002

Generic Name
AB-1002

AskBio's AB-1002 Gene Therapy Shows Promise in Heart Failure

• AskBio's AB-1002 gene therapy demonstrates preliminary efficacy in treating congestive heart failure, with no serious adverse events reported in early clinical data. • The Phase 1 study (NAN-CS101) showed clinically meaningful improvements in left ventricular ejection fraction (LVEF) in a subset of patients with NYHA Class III heart failure. • AB-1002 delivers a gene coding for inhibitor-1 (I-1), which blocks protein phosphatase 1 (PP1), a target implicated in the development of heart failure. • Bayer's AskBio is planning a Phase 2 trial to further evaluate the safety and efficacy of AB-1002 in a larger patient population.

AskBio's AB-1005 Gene Therapy for Parkinson's Disease Gains FDA RMAT Designation and Enrolls First Phase II Participants

• AskBio's AB-1005, a gene therapy for Parkinson's disease, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, potentially expediting its development. • The RMAT designation was supported by positive 36-month Phase Ib data, demonstrating a favorable safety profile and encouraging trends in clinical outcome measures. • The Phase II REGENERATE-PD trial, evaluating AB-1005 in moderate-stage Parkinson's, has randomized its first participants across sites in the US, with plans to expand to Europe. • AB-1005 delivers glial cell line-derived neurotrophic factor (GDNF) to promote the survival of dopaminergic neurons, addressing a critical unmet need in Parkinson's treatment.
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