Scholars@Duke
Deeksha Sarihyan Bali

Deeksha Sarihyan Bali

Professor of Pediatrics
Pediatrics, Medical Genetics
bali0001@duke.edu
4th Floor, GSRBI, 905 LaSalle Street, Division of Medical Genetics, Durham, NC 27710
801 Capitola Drive, Suite 6, Durham, NC 27713

Selected Grants

MPSIIIa Clinical CRIM

ResearchPrincipal Investigator · Awarded by Denali Therapeutics Inc. · 2025 - 2027

Phase 1 Study of In Utero Enzyme Replacement Therapy for the Treatment of Lysosomal Storage Diseases

ResearchInvestigator · Awarded by University of California - San Francisco · 2022 - 2026

Factors in Immune Response Affecting Long-term Treatment Outcomes in Pompe disease (CRIM)

Clinical TrialInvestigator · Awarded by Genzyme Corporation · 2022 - 2025

Liver-directed AAV gene therapy for PHKG2-Glycogen Storage Disease IX (GSD IX y2)

FellowshipMentor · Awarded by National Institutes of Health · 2021 - 2024

Factors in Immune Response Affecting Long-term Treatment Outcomes in Pompe disease (CRIM)

Clinical TrialInvestigator · Awarded by Genzyme Corporation · 2019 - 2021

Pharmacodynamic study using rhaGLU in a Pompe mouse model. Nonclinical GAA-KO mouse study design proposal rhaGLU

ResearchCo-Principal Investigator · Awarded by Pharming Group N.V. · 2019 - 2021

Development and validation of skin fibroblast Cross Reactive Immunological (CRIM) assay for MPSIIIa.

ResearchPrincipal Investigator · Awarded by Swedish Orphan Biovitrum SARL · 2018 - 2020

A Phase I Study of the Safety of AAV2/8 LSPhGAA in Late-onset Pompe Disease

Clinical TrialCo Investigator · Awarded by National Institutes of Health · 2017 - 2020

Potency Assays

ResearchInvestigator · Awarded by Actus Therapeutics · 2018 - 2019

Factors in Immune Response Affecting Long-term Treatment Outcomes in Pompe disease

Clinical TrialInvestigator · Awarded by Genzyme Corporation · 2016 - 2018

Activity and Biodistribution of the AAV2/8-LSPhGAA in GAA-knockout mice

ResearchCo Investigator · Awarded by Actus Therapeutics · 2018 - 2018

Blood spot GAA Enzyme Testing and GAA Gene Sequencing for Patients Suspected to have Pompe Disease

Public ServicePrincipal Investigator · Awarded by Genzyme Corporation · 2016 - 2017

A study to identify the frequency of lysosomal acid lipase deficiency in at-risk patient populations

ResearchPrincipal Investigator · Awarded by Synageva BioPharma · 2014 - 2016

Supplemental Funding for Phase 1/2 study of Clenbuterol for the Treatment of Pompe Disease

Clinical TrialCo Investigator · Awarded by Roivant Sciences, Inc. · 2015 - 2016

Phase 1/2 Study of Clenbuterol for the Treatment of Pompe Disease

Clinical TrialCo Investigator · Awarded by Food and Drug Administration · 2013 - 2016

Analysis of acide alpha-glucosidase (GAA) enzyme activity and full gene sequencing in the suspected US patient population

Public ServicePrincipal Investigator · Awarded by Genzyme Corporation · 2014 - 2016

Lab on a Chip for Multiplexed Newborn Screening of Lysosomal Storage Disease

ResearchPrincipal Investigator · Awarded by Baebies, Inc · 2009 - 2015

Measurement of LAL enzyme activity and LIPA gene sequencing on suspected Wolman and CESD disease patients

ResearchPrincipal Investigator · Awarded by Synageva BioPharma · 2013 - 2014

Clinical Trial Planning in Pompe Disease

ResearchCo Investigator · Awarded by National Institutes of Health · 2013 - 2014

Gene delivery to striated muscle by systemic AAV vectors

ResearchCo Investigator · Awarded by National Institutes of Health · 2006 - 2012