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ScholarsBetaWeill Cornell Medicine

Overview

Vaccines for addicitve drugs including cocaine and nicotine

Genetic vaccine for Pseudomonas aeruginosa

Gene therapy for alpha-1 antitrypsin deficiency

Gene therapy for diffuse coronary artery disease

Gene therapy for age-related macula degeneration and diabetic retinopathy

Gene therapy for glioblastoma and Ovarian Cancer

Gene Therapy approaches for Alzheimer's disease

Gene Therapy for Batten disease

Identification of biomarkers from the metabolome for Batten disease

Gene Therapy for Friedreich's Ataxia

Highlights

  1. 1
    Human gene therapy · 2020
    DSDolan SondhiRGRonald G CrystalSMStephen M. Kaminsky
    PMID: 32646255DOISenior author29 citations
  2. 2
    Human gene therapy · 2023
    DSDolan SondhiRGRonald G CrystalSMStephen M. Kaminsky
    PMID: 37171121PMCID: PMC10457653DOISenior author8 citations

Publications

89 publications

Funding

23 grantsView NIH portfolio on RePORTER ↗

Active

8 grants
  • PI
    Violet Research Institute
    Violet Research Institute · 2026–2031Contract
    VRI 264237-01
  • MPI
    MSA with University of Sheffield and Weill Cornell
    University of Sheffield · 2019–2030Contract
    SA 202209-02
  • Co-I
    Gene Therapy for Alpha 1-Antitrypsin Deficiency
    National Heart, Lung, and Blood Institute · 2024–2028
    R61HL169190-01A1
  • Co-I
    Gene Therapy for APOE4 Homozygous Alzheimer's Disease
    National Institute on Aging · 2026–2028
    UG3AG098024-01
  • MPI
    Phase IA/IB Study of AAVrh.10hFXN Therapy to Treat the Cardiomyopathy of Friedreich's Ataxia
    National Heart, Lung, and Blood Institute · 2020–2027
    R61HL151355-01
  • Sub-PI
    PC8.1-135: Integrating High-Throughput Chromatography and Data Science in a Platform for the Rapid and Accurate Quantification of Full, Partial, and Empty AAV Capsids
    National Institute of Standards and Technology · 2025–2027 · via University of Delaware
    70NANB21H086
  • Co-I
    Lexeo Biopharma Agreement
    LEXEO Therapeutics, Inc. · 2025–2027BioPharma Alliance
    BP 251293-02
  • PI
    MSA between Emily's Garden Foundation LTD and WCMC
    Emily's Garden Foundation LTD · 2026–2026Contract
    EGFL 260415-01
Completed grants (15)
  • Co-I
    Rapid Noninvasive Whole-body Imaging of AAV Gene Transfer Vectors
    National Institute of Biomedical Imaging and Bioengineering · 2019–2025
    R01EB027918-03
  • Co-I
    Manufacturing Services Agreement between PTC and WCM
    PTC Therapeutics · 2019–2024Contract
    SA 201427-01
  • MPI
    MSA with University of Pittsburgh and Cornell
    University of Pittsburgh · 2020–2024Contract
    SA 204534-01
  • Co-I
    Clinical Assessment of Anti-cocaine Vaccine dAdGNE in Cocaine Addicts
    National Institute on Drug Abuse · 2019–2024
    U01DA048524-01
  • PI
    AAV-Based Gene Therapy for the Long-Term Treatment of CLN2-Associated Retinal Degeneration and Prevention of Blindness in CLN2 Patients
    Daedalus Fund for Innovation (Weill Cornell) · 2021–2023BioPharma Alliance
    BP 214968-01
  • Co-I
    Gene Therapy for Cardiac Manifestations of Friedreich's Ataxia
    Friedreich's Ataxia Research Alliance · 2019–2021
    FARA 194278-01
  • Co-I
    Gene Therapy for Chronic Traumatic Encephalopathy
    Daedalus Fund for Innovation (Weill Cornell) · 2019–2021BioPharma Alliance
    DFI 200106-01
  • Co-I
    Persistent Gene Transfer to Mitochondria
    U.S. Department of Defense · 2017–2020
    W81XWH-17-1-0379
  • KP
    Disrupted Adenovirus Serotype 5-Based Anti-Cocaine Vaccine
    National Institute on Drug Abuse · 2012–2018
    U01DA033835-03
  • KP
    Mucosal Immunization Against P. aeruginosa by Modified Adenovirus Vectors
    National Institute of Allergy and Infectious Diseases · 2013–2018
    R01AI103341-03
  • KP
    Second-Generation Gene Transfer Vector-Based Anti-Cocaine Vaccines
    National Institute on Drug Abuse · 2013–2017
    R01DA032702-03
  • Sub-PI
    Final Preclinical Development of Gene Therapy for Post-Operative Atrial Fibrillation
    National Institute on Aging · 2014–2017 · via University of Massachusetts Medical Center
    R21AG042701-02 EW
  • MPI
    Adenovirus-Based Nanoparticle Vaccines
    National Institute on Drug Abuse · 2009–2014
    R01DA025305-05
  • MPI
    Preclinical Development of Genetically-Delivered Bevacizumab for Treatment of Wet Macular Degeneration
    National Eye Institute · 2013–2014 · via REGENX Bio, Inc.
    R43EY023108-01 EW
  • KP
    Adenovirus-Based Anti-Cocaine Vaccine
    National Institute on Drug Abuse · 2009–2011
    RC2DA028847-02 EW

External relationships

Ownership

LEXEO Therapeutics, Inc.

Proprietary Interest

LEXEO Therapeutics, Inc.

Other

Renova Therapeutics