Kasturi Sengupta
Wayne,PA
Summary
A distinguished senior scientist was acknowledged for outstanding productivity and efficiency within a fast-paced environment. Extensive expertise in gene therapy, emphasizing liver, muscle, and CNS. Proficient in study design, data analysis and supervising team. Strong analytical abilities and problem-solving expertise cultivate effective teamwork and lead to project success.
Overview
8
8
years of professional experience
Work History
Senior Scientist
Code Biotherapeutics
10.2022 - 05.2025
- Lead research programs to develop muscle- and liver-targeted non-viral gene therapy.
- Accomplished PoP, PoC in vivo studies to evaluate DNA nanoparticle-driven payload delivery to target tissues.
- Established optimized workflows using ddPCR, ddRT-PCR, and Simple Western (Jess), and multiplex IHC to enable sensitive and high-throughput detection of diverse payloads and protein markers in cells and tissues.
- Supervised a team for executing cell-based assays (transfection, transduction, receptor binding, payload release, and delivery), flow cytometry, ICC, confocal imaging, and facilitating proof-of-concept mechanistic insight into drug delivery.
- Enabled data-driven decision-making by performing robust quantitative analyses to guide lead candidate selection for preclinical pipelines.
- Built and maintained productive collaborations with academic institutions and industry partners to accelerate research goals.
Senior Research Investigator
University of Pennsylvania
07.2020 - 09.2022
- Led cross-functional teams in conducting preclinical proof-of-concept studies across muscle and CNS disease models, contributing to key therapeutic advancements.
- Designed and characterized novel AAV vectors with unique microdystrophins, driven by striated muscle-specific promoters, to assess the functionality and therapeutic efficacy of novel microdystrophin vectors for Duchenne muscular dystrophy gene therapy.
- Evaluated and selected optimal promoters for efficient and tissue-specific gene expression in cardiac, diaphragm, and skeletal muscle models, supporting vector design for therapeutic development.
- Co-led an AAV-based gene therapy initiative for Angelman Syndrome, engineering vectors for neuron-specific delivery of UBE3A. Directed biochemical and image-based quantification strategies, including Visiopharm software for high-resolution analysis of UBE3A expression at the single-neuron level in targeted brain regions.
- Coordinated with multiple teams, including AAV manufacturing, for NHP toxicity and biodistribution studies, the development of sensitive qPCR assays, and the interpretation of clinical pathology to support IND-enabling research.
- Executed precise stereotaxic surgeries for targeted AAV administration in the mouse brain, supporting CNS-focused preclinical studies.
Post-Doctoral Research
University of Pennsylvania
01.2017 - 06.2020
- Developed a novel CRISPR/Cas9 genome editing strategy in DMD-patient-derived iPSCs to prevent miRNA-mediated repression of utrophin expression. Functional validation in genome-edited and differentiated myoblasts as proof of concept.
- Designed, tested, and optimized morpholino oligo-based site-blocking oligos (SBO) to inhibit let-7c miRNA binding to the utrophin UTR.
- Designed and screened SBOs with different chemical structures for the identification of the highest inhibition of miRNA binding to the target gene.
- Proof of concept validation of the lead candidate in a preclinical model.
- Selected to present research findings at scientific conferences, demonstrating strong communication skills, and recognition of research impact.
Education
PhD - Microbiology
University of Calcutta
India03-2014
Skills
- Cell-based assay design
- qPCR, ddPCR
- Jess
- Flow
- IHC
- Confocal
- Target screening for receptor-mediated payload delivery
- Analytical method development for DNA nanoparticle characterization
- CRISPR-based genome editing
- SOP and study report
- Scientific data presentation
- Supervising team
- Collaboration with external collaborator
Selected Presentations And Publications
- Genome editing-mediated utrophin upregulation in Duchenne muscular dystrophy stem cells, Sengupta K. et al…. Khurana T.S, Molecular Therapeutic Nucleic Acids, 22, 500-509, 2020, https://scholar.google.com/citations?view_op=view_citation&hl=en&user=P0XO194AAAAJ&citation_for_view=P0XO194AAAAJ:UeHWp8X0CEIC
- PMO-based let-7c site blocking oligonucleotide (SBO) mediated utrophin upregulation in mdx mice, a therapeutic approach for Duchenne muscular dystrophy (DMD), Sengupta K. et al…..Khurana T.S, Scientific Reports, 10, 21492, 2020, https://scholar.google.com/citations?view_op=view_citation&hl=en&user=P0XO194AAAAJ&citation_for_view=P0XO194AAAAJ:IjCSPb-OGe4C
- Functional improvement of dystrophic muscle by repression of utrophin: let-7c interaction, Mishra M.K, Loro E, Sengupta K. et al.. & Khurana T.S, PLoS One, 12, e0182676, 2017, https://scholar.google.com/citations?view_op=view_citation&hl=en&user=P0XO194AAAAJ&citation_for_view=P0XO194AAAAJ:9yKSN-GCB0IC
- CRISPR/Cas9 genome editing of Utrophin miRNA-binding sites in DMD patient hiPSCs, New Direction in Biology and Diseases of Skeletal Muscle Conference, 2018, Poster presentation
- A microbiome-dependent gut–brain pathway regulates motivation for exercise, Dohnalová et al, Nature, 612, 739-747, 2022, https://scholar.google.com/citations?view_op=view_citation&hl=en&user=P0XO194AAAAJ&citation_for_view=P0XO194AAAAJ:Y0pCki6q_DkC
- Gene Therapy-Mediated Cross Correction for CDKL5 Deficiency Disorder, Percival J, Born H, Sengupta K. et al., Molecular Therapy, 29, 4, 83-83, 2022, Invited talk at ASGCT
- Gene Replacement Therapy for Angelman Syndrome, Percival J, Sengupta K, et al., Molecular Therapy, 29, 4, 83-83, 2021, Presented at ASGCT. Invited talk at ASGCT.
- Gene therapy-mediated cross correction for CDKL5 deficiency disorder, 2021 CDKL5 Forum. Annual Meeting of the Loulou Foundation, 11/01/21, Invited Talk
- Gene Replacement Therapy for Angelman Syndrome, Annual Meeting of the Foundation for Angelman Syndrome Therapeutics (FAST), Translational Research Symposium, 12/05/20, Invited Talk
- High-throughput identification of post-transcriptional utrophin up-regulators for Duchenne muscle dystrophy (DMD) therapy, Loro E, Sengupta K. et al.. Khurana T.S, Scientific Reports, 10, 4039, 2020, https://scholar.google.com/citations?view_op=view_citation&hl=en&user=P0XO194AAAAJ&citation_for_view=P0XO194AAAAJ:2osOgNQ5qMEC
Timeline
Senior Scientist
Code Biotherapeutics
10.2022 - 05.2025
Senior Research Investigator
University of Pennsylvania
07.2020 - 09.2022
Post-Doctoral Research
University of Pennsylvania
01.2017 - 06.2020
PhD - Microbiology
University of Calcutta
Similar Profiles
EMILY KALIN, CPAEMILY KALIN, CPA
Senior Financial Reporting Analyst at SAB BiotherapeuticsSenior Financial Reporting Analyst at SAB Biotherapeutics
Katrina A. ComberKatrina A. Comber
Business Administrator at Aro BiotherapeuticsBusiness Administrator at Aro Biotherapeutics
EDWARD TERRAZASEDWARD TERRAZAS
Quality Control Associate /Quality Control Microbiologist at A2 BiotherapeuticsQuality Control Associate /Quality Control Microbiologist at A2 Biotherapeutics
Colin CostinColin Costin
Anti Money Laundering Investigator at Robert HalfAnti Money Laundering Investigator at Robert Half
Zachary BarbarisiZachary Barbarisi
Server at Mercato RistoranteServer at Mercato Ristorante