About our CDKL5 AAV9 program

A. Personal Statement

His leadership in a phase I gene therapy trial for AADC deficiency, a condition similar to CDKL5 deficiency due to loss of functional protein, equips him to oversee study design, in vivo surgical delivery, and coordination with the research team. Dr. Gupta's expertise ensures the preclinical development of the AAV9.Syn.hCDKL5 vector will meet rigorous safety and efficacy standards, advancing toward FDA IND submission. His role as Principal Investigator of the UCSF Brain Tumor Research Center and his involvement in national clinical studies further underscore his capability to drive this project forward.

B. Positions and Honors

Positions:

• 2000–Present: Professor, Departments of Neurological Surgery and Pediatrics, UCSF
• 2000–Present: Chief, Division of Pediatric Neurosurgery, UCSF
• 2000–Present: Principal Investigator, Brain Tumor Research Center, UCSF
• 1998–2000: Clinical Associate, Section of Neurosurgery, University of Chicago

Honors:

• TP Morley Resident Research Award, University of Toronto
• Multiple teaching awards during residency, University of Toronto

C. Contributions to Science

1. Advancements in Fetal Surgery for Myelomeningocele

Dr. Gupta led the neurosurgical component of the Management of Myelomeningocele Study (MOMS), demonstrating that prenatal surgery reduces the need for shunting and improves motor outcomes compared to postnatal repair. This work has established prenatal surgery as a standard of care for selected cases.

• Adzick, N. S., et al. (2011). A Randomized Trial of Prenatal versus Postnatal Repair of Myelomeningocele. New England Journal of Medicine, 364(11), 993–1004. [NEJM]

2. Pioneering Gene Therapy for Neurological Disorders

As clinical Principal Investigator, Dr. Gupta contributed to a phase I study evaluating gene therapy for AADC deficiency, showing improvements in motor and cognitive functions via AAV2-hAADC delivery to the midbrain. This experience is directly applicable to the CDKL5 project.

• Pearson, T. S., et al. (2021). Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons. Nature Communications, 12, 4251. [Nature]

3. Research on Brain Tumor Progression

Dr. Gupta's laboratory investigates inflammation's role in pediatric brain tumor progression, particularly medulloblastoma, identifying novel therapeutic targets to improve treatment outcomes.

• Chinnam, D., et al. (2023). Immunohistochemical Surrogates for Molecular Stratification in Medulloblastoma. Applied Immunohistochemistry & Molecular Morphology, 31(8), 561–568. [PubMed]

D. Research Support

Ongoing:

• NIH R01CA246722 (07/01/2020–06/30/2025): Myeloid cells and radiation-induced memory deficits in rodent glioma model: sex and age effects. Role: Co-Principal Investigator.

Completed:

• V Foundation 02016-007 (04/15/2016–10/15/2019): Convection-enhanced delivery of nanoliposomal CPT-11 for DIPG. Role: Co-Investigator.
• NIH K08NS055061 (04/19/2006–02/28/2011): Macrophage and Microglial Activation in Glioma-Associated Inflammation. Role: Principal Investigator.

A. Personal Statement

Her career includes significant achievements in advancing therapeutic candidates through preclinical development to IND submission. At Pfizer, Dr. Patel spearheaded non-human primate (NHP) studies to develop immunosuppression strategies for AAV gene therapy, addressing critical barriers to treatment success. She also led a biology team in transitioning a recombinant protein program to the IND filing stage, showcasing her regulatory expertise. At Homology Medicines, Inc., she drove a neuroscience gene therapy program from exploratory research to IND readiness, conducting proof-of-concept studies that led to a clinical candidate nomination. For the CDKL5 gene therapy project, Dr. Patel will apply her expertise to optimize the immunological profile of the AAV9.Syn.hCDKL5 vector, designing studies to minimize adverse immune responses and maximize therapeutic benefit. Her skills in assay development, immunogenicity assessment, and cross-functional leadership are critical to achieving FDA IND submission and advancing treatment for CDKL5 deficiency disorder (CDD).

B. Positions and Honors

Positions:

• 2023–Present: Associate Director, AAV Immunology, Solid Biosciences
• 2022–2023: Principal Scientist, Pfizer
• 2020–2022: Scientist II, Homology Medicines, Inc.
• 2018–2020: Scientist I, Homology Medicines, Inc.
• 2017–2018: Postdoctoral Fellow, Broad Institute of MIT and Harvard
• 2016–2018: Research Fellow, Harvard Medical School
• 2011–2016: Ph.D. in Molecular and Translational Medicine, Boston University School of Medicine

Honors:

• Harvard Business School Leadership Essentials Program, 2023

C. Contributions to Science

1. Development of Gene Therapy for Hunter Syndrome

Dr. Patel contributed to HMI-203, an investigational gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome. Her work involved preclinical studies to evaluate efficacy and safety, advancing the therapy toward clinical trials.

• Patel, K. R., et al. (2021). HMI-203: Investigational gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome. Molecular Genetics and Metabolism, 132(2), S82. [ResearchGate]

2. Leadership in Preclinical Gene Therapy Development

At Homology Medicines, Inc., Dr. Patel led IND-enabling studies for a neuroscience gene therapy program. She developed in vitro and tissue-based assays for target validation and proof-of-concept studies, successfully nominating a clinical candidate and advancing it to the IND stage through cross-functional collaboration.

3. Advancements in Immunosuppression for Gene Therapy

At Pfizer, Dr. Patel led NHP studies to identify immunosuppression strategies for AAV gene therapy platforms. Her work addressed immune responses that limit treatment efficacy, designing and analyzing experiments to enhance therapy safety and effectiveness.

4. Research on Neuro-Immune Interactions

As a postdoctoral fellow at the Broad Institute and Harvard Medical School, Dr. Patel investigated microglia's role in Alzheimer's disease pathology. She developed high-throughput screening methods to study gene functions related to amyloid-beta uptake, enhancing understanding of neuro-immune crosstalk in neurodegenerative disorders.

A. Personal Statement

Dr. Gingras's breadth of experience in therapeutic development, strategic program planning, and scientific innovation helps ensure that our approach to CDKL5 gene therapy is informed by both rigorous science and practical pathways toward clinical translation.

A. Personal Statement

Paul has held senior leadership roles at AavantiBio, BridgeBio, LogicBio Therapeutics, and Pfizer, where he built and scaled cGMP manufacturing platforms to support IND and clinical programs. His expertise includes CMC strategy, regulatory readiness, and the production of high-quality clinical-grade AAV vectors. For the CDKL5 gene therapy program, Paul's experience is critical to ensuring scalable, compliant, and reliable vector manufacturing. His leadership helps bridge early scientific innovation with real-world clinical delivery for patients and families.

A. Personal Statement

At Bayer Corporation, Dr. Bhargava led cross-functional preclinical teams and contributed to projects that progressed toward Phase I clinical development, demonstrating deep experience in translational research and regulatory readiness. His leadership in assay design, model development, and preclinical strategy provides valuable support to the CDKL5 gene therapy program by strengthening our ability to generate robust, IND-supporting preclinical data and to efficiently address key scientific questions as we advance toward clinical milestones.

A. Personal Statement

At AskBio, as Senior Director of Innovation, he led R&D efforts for AAV-based gene therapies, building on the pioneering work of Dr. Richard Jude Samulski. His expertise spans viral vector design, preclinical animal models, and translational research, as demonstrated in his work on hepatitis and oncolytic virotherapies. As a member of the ARDAT consortium and the Dravet Syndrome Foundation Spain's Scientific Evaluation Board, Dr. Suarez has shaped gene therapy strategies for rare diseases. For the CDKL5 gene therapy project, Dr. Lester Suarez has done gene therapy vector development, vector delivery optimization and preclinical testing, ensuring the therapy's efficacy and safety. Dr. Suarez's contributions help bridge the gap between preclinical studies development and clinical application.

B. Positions and Honors

Positions:

• 2024–Present: Vice President, Research and Development, M34, Inc., North Carolina, USA
• 2024–Present: Affiliate Faculty, CSU College of Veterinary Medicine and Biomedical Sciences, Fort Collins, Colorado, USA
• 2020–2025: Member, Scientific Evaluation Board, Dravet Syndrome Foundation Spain, Spain
• 2021–2024: Member, ARDAT Consortium
• 2023–2024: Senior Director of Innovation, Asklepios BioPharmaceutical, Inc. (AskBio), Raleigh-Durham, North Carolina, USA
• 2022–2023: Director of Innovation, AskBio, North Carolina, USA
• 2019–2022: Director, Research & Development, AskBio, Raleigh-Durham, North Carolina, USA
• 2019: Senior Research Scientist, AskBio, Raleigh-Durham, North Carolina, USA
• 2018–2019: Postdoctoral Research Associate, UC San Diego School of Medicine, La Jolla, California, USA
• 2015–2018: Postdoctoral Research Associate, Salk Institute for Biological Studies, La Jolla, California, USA
• 2011–2015: Graduate Research Teaching Assistant, University of Navarra, Pamplona, Navarra, Spain
• 2010–2015: Researcher PhD Student, Cima Universidad de Navarra, Pamplona, Navarra, Spain
• 2014: Visiting PhD Student, University of Heidelberg, Heidelberg, Germany
• 2006–2009: Research Assistant Graduate Student, Centro de Ingeniería Genética y Biotecnología, Havana, Cuba

C. Contributions to Science

1. Advancements in AAV Gene Therapy

At AskBio, Dr. Suarez led innovation in AAV-based gene therapies for rare diseases, optimizing viral vectors for enhanced delivery and efficacy. His work contributed to the advancement of therapeutic candidates toward clinical trials, building on AskBio's pioneering AAV platform.

2. Preclinical Models for Viral Therapies

During his postdoctoral research at the Salk Institute and UC San Diego, Dr. Suarez developed preclinical models for oncolytic virotherapies and flavivirus infections. His studies on adenovirus and molecular virology provided insights into host-pathogen interactions, informing therapeutic design.

3. Gene Therapy for Viral Hepatitis

As a PhD student at Cima Universidad de Navarra and a visiting researcher at the University of Heidelberg, Dr. Suarez investigated HBV and HDV using animal models and Trojan horse virus strategies. His work advanced gene therapy approaches for viral hepatitis, contributing to novel therapeutic strategies.

4. Therapeutic Cancer Vaccines

At the Centro de Ingeniería Genética y Biotecnología, Dr. Suarez developed viral vector-based cancer vaccines using transgenic mice models. His research laid the groundwork for innovative immunotherapies targeting cancer.

A. Personal Statement

As founder and CEO of Coté Orphan LLC, he grew a boutique firm that facilitated regulatory approvals for rare disease therapies, serving over 400 clients before its sale to QuintilesIMS in 2017. Currently, as CEO of Only Orphans Coté LLC and Partner at Porcine Preclinical Partners, Dr. Coté leverages his expertise to advance preclinical and regulatory strategies for innovative therapies. An anatomic pathologist and medical epidemiologist, he has authored 80 peer-reviewed articles on topics including HIV/AIDS-related malignancies and public health policy. For the CDKL5 gene therapy project, Dr. Coté will guide regulatory submissions, ensuring the AAV9.Syn.hCDKL5 vector meets FDA standards for IND submission, addressing the critical unmet needs of CDKL5 deficiency disorder (CDD) patients.

B. Positions and Honors

Positions:

• 2021–Present: CEO, Only Orphans Coté LLC, Cambridge, MA and Washington, DC, USA
• 2022–Present: Partner, Porcine Preclinical Partners, Cambridge, MA and Ames, IA, USA
• 2017–2021: Independent Entrepreneur, Self Employed, Washington DC-Baltimore Area, USA
• 2017: Vice President, Quintiles (now IQVIA), Silver Spring, MD, USA
• 2012–2017: Principal & CEO, Coté Orphan LLC, Silver Spring, MD, USA
• 2011–2014: Professor of Regulatory Practice, Keck Graduate Institute, Claremont, CA, USA
• 2011–2012: Chief Medical Officer, National Organization for Rare Disorders, USA
• 2007–2011: Director, FDA Office of Orphan Products Development, FDA, USA
• 2006–2007: CDC Country Director for Rwanda, Centers for Disease Control and Prevention, Rwanda
• 2004–2006: Senior Federal Advisor to the Director, District of Columbia Department of Health, USA
• 2002–2004: Branch Chief, Therapeutics and Blood Safety, CBER, FDA, USA
• 1999–2002: Medical Director, Cancer Statistics Branch, National Cancer Institute, Rockville, MD, USA
• 1998–1999: Medical Epidemiologist (PHS Detail), USDA/FSIS, USA
• 1995–1998: Resident in Anatomic Pathology, National Institutes of Health, Bethesda, MD, USA

C. Contributions to Science

1. Advancement of Orphan Drug Development

As Director of FDA's OOPD, Dr. Coté implemented the Orphan Drug Act, overseeing decisions on over 1,400 orphan drug designations. His leadership expanded therapeutic options for rare diseases, including neurological disorders like CDKL5 deficiency.

2. Regulatory Innovation for Rare Diseases

Through Coté Orphan LLC, Dr. Coté facilitated regulatory approvals for numerous rare disease therapies, managing submissions to FDA and EMA. His firm's work supported a significant share of orphan designations, accelerating treatments to market.

3. Epidemiological Research on Public Health

Dr. Coté authored 80 peer-reviewed articles, including studies on HIV/AIDS-related malignancies, typhoid fever epidemics, and bicycle helmet laws' impact on injury statistics. His work has informed public health policy and clinical practice.

4. Cancer Surveillance and Data Systems

As Medical Director at NCI's Cancer Statistics Branch, Dr. Coté oversaw the SEER registry, managing data from over 2 million cancer cases. He developed POPBLOCKS, the first population-based tissue collection system, enhancing cancer research capabilities.