Allison Knoll, PhD
Strategic Consultant
About
Translational neuroscience leader with 15+ years advancing CNS therapeutics from discovery through IND clearance. Most recently led preclinical and IND-enabling strategy for a first-in-class AAV gene therapy for STXBP1 Encephalopathy at Capsida Biotherapeutics. Core expertise: - Preclinical strategy and IND-enabling research for CNS disorders - Translational neuroscience: behavioral endpoints, disease models, biomarker development - Gene therapy development: AAV, GLP toxicology, NHP studies, regulatory strategy - Cross-functional leadership: led teams through discovery, preclinical, and regulatory milestones Currently exploring: - Consulting opportunities in preclinical strategy for CNS gene therapies and emerging therapeutics - Full-time leadership roles (remote or LA-based) in translational neuroscience and drug development - Applications of AI in neuroscience-informed drug discovery Passionate about rare pediatric diseases and committed to advancing treatments for neurodevelopmental and CNS disorders.
United States
Calabasas
Research
Strategic Program Development, Therapeuitic Modality Strategy, Scientific Advisory Board Leadership, KOL Engagement, Gene Therapy, Preclinical Development, Cross-functional Team Leadership, Central Nervous System Disorders, High Throughput Screening, Molecular Biology, Neuroscience, Cell Biology, Biochemistry, Life Sciences
Experience
Strategic Consultant
Rare Disease Foundation (confidential)
United States
Advising a rare disease foundation on therapeutic strategy, translational readiness, and development priorities for a rare neurodevelopmental disorder program. Shaping the scientific and program decisions that advance an urgent family-led mission toward biotech partnership and IND. Leading therapeutic modality review, preclinical gap analysis, and expert interviews. Working closely with foundation leadership on the decision decks, strategic briefs, and SAB sessions that shape funding, modality, and roadmap decisions.
Director Preclinical Research
Capsida Biotherapeutics
Thousand Oaks, California, United States
Led preclinical and IND-enabling strategy for CAP-002, a first-in-class AAV gene therapy for STXBP1 encephalopathy, advancing the program from discovery through IND clearance and contributing to Capsida's transition from startup to clinical-stage biotech. - Directed preclinical strategy in partnership with CSO; led team of 6 scientists and co-led cross-functional group of ~15 within matrixed R&D organization - Oversaw outsourced preclinical studies including NHP dose-range finding, GLP toxicology, iPSC efficacy validation, and potency assay development - Led preclinical regulatory response strategy; developed FDA briefing documents for INTERACT and pre-IND meetings; supported Orphan Drug and Fast Track designations - Collaborated across clinical, CMC, and regulatory functions to deliver IND-ready preclinical packages for CNS-targeted AAVs - Represented Capsida at STXBP1 Foundation meetings, strengthening industry-patient partnerships - Built high-performance team culture rooted in scientific rigor, openness, and innovation
Associate Director, Preclinical Research
Capsida Biotherapeutics
Thousand Oaks, California, United States
Research Assistant Professor
University of Southern California
Greater Los Angeles Area
Postdoctoral Research Fellow
Children's Hospital Los Angeles (CHLA)
Postdoctoral Fellow
University of Southern California
Los Angeles, CA
Postdoctoral Fellow studying the development of social behavior
Research Affiliate
Broad Institute of MIT and Harvard
Cambridge, MA
Worked on a high-throughput drug screen as an affiliate with the Chemical Biology Platform.
Graduate Student Researcher
Harvard University
Belmont, MA
Studied the neurobiology of fear and anxiety in rats.
Education
Harvard University
Neurobiology
University of Wisconsin-Madison
Biochemistry & Molecular Biology
Allison Knoll, PhD's Contact Information
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