Preclinical and Translational Research Webinar Series

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Introduction

Odylia Therapeutics and the Foundation Fighting Blindness have teamed up to hold a four (4) part preclinical and translational research webinar series. This series will focus on genetic technologies for blinding diseases and will provide tangible advice and educational materials to researchers in the inherited retinal disease (IRD) space as well as the broader rare disease field.

Both Odylia and the Foundation Fighting Blindness are focused on supporting the research community in developing treatments and cures for inherited retinal dystrophies. The world has seen a gene therapy for IRDs reach the pinnacle of success with the 2017 FDA approval of LUXTURNA. Although this success has opened the door for other IRD gene therapies, there is still a multitude of genetically-driven diseases in this space that require a robust pipeline for therapeutic development.

This webinar series will provide actionable preclinical development information to researchers looking to advance their ideas into the clinic to help patients.

If you are currently working in or thinking about preclinical development, please join us for this engaging webinar series.

Webinar Format

  • All webinars will be virtual and recorded for later viewing
  • Invited speakers will give a detailed presentation covering preclinical development topics, providing information to researchers to help shape their own projects/programs
  • Each webinar will have a Q&A session enabling researchers to bring their personal questions and issues to the table for discussion. Please send advance questions, if applicable, to either events@odylia.org or grants@fightingblindness.org (Please put “Preclinical Webinar Question” in the subject line)

Webinar Series

  • Webinar #1: Tools for Preclinical Development: Creating a Target Product Profile and Effectively Engaging Regulatory Entities
  • Webinar #2: Disease relevant translation models in ocular drug development: Types, challenges, advantages, and alternatives
  • Webinar #3: Working with Contract Research Organizations (CROs) and Contract Manufacturing Organizations (CMOs) to accelerate development
  • Webinar: #4: Attracting funding opportunities and a look towards the future of ocular drug development

Webinar #1

Tools for Preclinical Development: Creating a Target Product Profile and Effectively Engaging Regulatory Entities (October 2020).

Translating a potential therapy from the bench to the clinic takes an enormous amount of time, effort, and funds to achieve.  Therefore, it is paramount that those working to this end should work as efficiently as possible.  Early regulatory engagement can help streamline drug development.  One guiding tool that can help this process is the development of a Target Product Profile (TPP). The purpose of a TPP is to provide an easy to follow format for discussions between the research sponsor and the Food and Drug Administration (FDA). This document will help guide therapy development from pre-IND through post-marketing programs. Effective preparation of the TPP will allow for (1) the exclusion of the need to introduce the history of the drug development program(s), (2) enhance the sponsors interactions with FDA staff, (3) guiding pre-new drug application (pre-NDA) meetings ensuring the goals are aligned with sponsor’s official NDA submission, and (4) overall the TPP is used to update the FDA during engagements.

We want our research community to begin their preclinical efforts with their end goal in mind. This webinar will allow our community to learn about and actively discuss the best way to create a TPP and engage with regulatory agents to effectively move their research from the lab to the clinic.

Below is a recording of the webinar.

Webinar #1 Panelists:

Stephen Brenner
  • Stephen Brenner
    • Stephen is a consultant with more than 40 years of experience in the academic, government, and industrial sectors applying innovative chemical and protein technology to drug discovery. Dr. Brenner is a former VP of Chemical and Protein Technologies at Bristol-Myers Squibb and the former Senior Director of Physical Sciences, DuPont Pharmaceutical Company.
Suzanne Thornton-Jones
  • Suzanne Thornton-Jones 
    • Suzanne has a Ph.D. in Pharmacology and Toxicology from the Medical College of Virginia/Virginia Commonwealth University. In her current role at the Gene Therapy Program at the University of Pennsylvania, she leads the Regulatory Affairs department in developing the regulatory strategy for engagement of Health Authorities throughout the preclinical research, clinical trial, and registration processes.  In addition to her regulatory responsibilities, she also leads the Compliance group, Good Laboratory Practice (GLP) Quality Assurance Unit, which is responsible for auditing preclinical studies used to support Health Authority regulatory filings. Prior to joining GTP, Suzanne worked in Regulatory Affairs for 12 years in the Pharmaceutical industry at Sanofi and AbbVie, and 6 years as a pharmacology/toxicology reviewer at the Food and Drug Administration, Center for Drug Evaluation and Research (FDA/CDER).

Webinar #2

Disease relevant translation models in ocular drug development: Types, challenges, advantages, and alternatives (November 2020).

Every potential retinal disease therapy, whether small molecule, cell therapy, or genetic technology, will require significant assay development and disease model testing before being considered for clinical use. Over the past half century, the life sciences have seen a technological explosion that enables the research community to model and manipulate biological systems, which has created an expansive tool kit for preclinical development. However, the selection of the right tool for your technology and disease indication can be difficult at times, and if not planned out correctly, can lead to program delays.

This deep-dive webinar highlighted the advantages and disadvantages of certain retinal disease models, and their utility as tools to move new therapies toward Investigational New Drug (IND) filing and clinical use.

Below is a recording of the webinar.

Webinar #2 Panelists:

Neena Haider
  • Neena Haider, Associate Professor, Harvard University
    • Dr. Haider is a visionary and creative leader bridging medical sciences and healthcare, developing breakthrough therapies that have global impact. Neena received a Ph.D. from the University of Iowa in Genetics, part of the human genome project and involved in identifying over a dozen human disease associated genes. Dr. Haider directs a cutting-edge genetics and gene therapy laboratory at Harvard Medical School, developing and identifying viable therapeutic options for blindness such as retinitis pigmentosa, aged macular degeneration, and diabetic retinopathy bringing a product from bench to bedside. Her team is the first to develop a novel broad-spectrum gene therapy to treat multiple forms of blindness and has a U.S. patent issued and two FDA approval for orphan disease designations (ODD) gene therapy.
Kaylan Dulla
  • Kaylan Dulla, Senior Director Ophthalmology Non-Clinical Research, ProQR Therapeutics
    • Dr. Dulla leads and manages multiple highly complex projects and programs encompassing the development of Ophthalmology Franchise’s NMEs from inception to clinic, with major focus on pharmacology, translational and preclinical science.
Gustavo Aguirre
  • Gustavo Aguirre
    • Dr. Aguirre is Professor of Medical Genetics and Ophthalmology at The School of Veterinary Medicine, University of Pennsylvania. His work focuses on identifying the genetic causes of inherited blindness, identifying the mechanisms linking mutation to disease, and developing treatment approaches. Studies are done in the dog model which is affected a large variety of inherited photoreceptor diseases that are now being characterized at the molecular level. He earned his undergraduate, veterinary, and doctoral degrees at the University of Pennsylvania, where he also completed a residency in ophthalmology in the School of Veterinary Medicine before serving as a post-doctoral fellow at the Wilmer Ophthalmological Institute of the Johns Hopkins University School of Medicine. His work has been supported by the National Institutes of Health, the Foundation Fighting Blindness, and The Seeing Eye, Inc. He is an elected Fellow of the Association for Research in Vision and Ophthalmology, the College of Physicians of Philadelphia, and is a member of the Institute of Medicine of The National Academies.

Webinar #3

Working with Contract Research Organizations (CROs) and Contract Manufacturing Organizations (CMOs) to accelerate development (January 2021).

Repeatability of data and outcomes are some of the hardest things to achieve in research.  However, independent validation of data and outcomes are cornerstones to preclinical therapy development.  This is often achieved by working with Contract Research Organizations (CROs). The Food and Drug Administration defines a CRO as a person or entity that assumes, as an independent contractor with the sponsor, one or more of the obligations of a sponsor, e.g., design of a protocol, selection or monitoring of investigations, evaluation of reports, and preparation of materials to be submitted to the Food and Drug Administration. In addition to CROs, as a therapy moves toward clinical use it will need to be produced under current good manufacturing practice (cGMP) requirements before being considered for use in humans. Contract manufacturing organizations (CMOs), also called contract development and manufacturing organizations (CDMOs), are companies that provide drug development and  drug manufacturing services in the pharmaceutical industry on a contract basis.

In this deep-dive webinar, panelists discussed the ins and outs of working with CROs and CMOs providing a clear view of the capabilities and the expectations when seeking their services.

Below is a recording on the webinar:

Webinar #3 Panelists:

Image of Michelle Berg
  • Michelle Berg, President, GMP Nucleic Acids, Aldevron
    • Michelle Berg brings over 20 years of experience in the biotechnology sector, 10 years of which have been spent in executive leadership roles. In her current position as President of Aldevron’s GMP Nucleic Acids Business Unit, Berg oversees Aldevron’s strategy to provide GMP plasmids and mRNA for gene editing, gene therapy, and cell therapy applications. She works closely with the company’s operational team to meet the requirements of these growing fields, while supporting the clinical and commercial efforts of Aldevron’s clients. Aldevron’s GMP plasmid facility, located in Fargo, N.D., is the largest in the world. Berg earned a Bachelor of Science in Biotechnology from North Dakota State University (NDSU). She is a contributing author and speaker on patient-focused programming, rare disease advocacy, and accessible education on genetic medicines. In addition to her roles with Abeona and Aldevron, she has performed research on behalf of the Department of Plant Sciences at NDSU.
Image of Tom Foti
  • Tom Foti, President of the Protein Business Unit, Aldevron
    • Thomas G. Foti is the President of the Protein Business Unit at Aldevron and is based out of Madison, WI Operation. He has been in this position since May, 2019. Starting in 2009, Thomas was one of the original founders of Aldevron’s Protein Services Business. Over a period of 10 years, prior to working for Aldevron, he served in several roles in Merck KGaA’s Bioscience Division, most recently as the Director of its Global Custom Services Business. He started his career in 1992, with Novagen, Inc. serving in manufacturing and operational roles until 1999. He played college basketball while earning a Bachelor’s of Science in Biotechnology and Microbiology from North Dakota State University. He holds a Master’s in Business Administration from Edgewood College and a Management Leadership Certificate from Massachusetts Institute of Technology. Thomas has served on the regional Board of Directors for Thrivent Financials and the United Way Annual Campaign Cabinet. Thomas lives in Madison, WI with his wife Brenda and three daughters, Samantha, Brianna & Gabriella.
Image of Leslie Lemki-Boutcher
  • Leslie Lemke-Boutcher, Drug Development Leader, Covance
    • Leslie Lemke-Boutcher is a Board Certified Toxicologist (DABT) with over 20 years’ experience developing ocular drugs. She began her career in the pharma sector supporting projects from first target identification, through candidate selection, IND enabling program design/conduct, proceeding through NDA/BLA submission and global regulatory submission defense.  In her role at Covance, Leslie leads a group of professional drug developers who like her, bring the benefit of their comprehensive experience to clients who are looking for scientific and strategic guidance to optimize their nonclinical and translational programs.

Webinar #4

Funding & Execution of Ocular Drug Development (March 2021).

There are approximately 40 clinical trials aimed at addressing inherited retinal disease and dry Age-Related Macular Degeneration, along with emerging technologies in the preclinical development pipeline. As therapies move from the bench to the bedside, proper funding and management are critical elements in the technology’s journey to the patient population. Financial support comes in many forms, such as venture capital, government programs, or industry to name a few. However, it is paramount that academics align their study designs and goals with these support systems to adequately secure and execute these funds. The other side of the coin is entrepreneurship, management, and oversight. While the technology must address its target disease effectively and safely, ample leadership is imperative to guide and championing the asset as it navigates the path to clinical use.   

Here, in Webinar #4, we bring you three experts on venture capital funding, Small Business Innovation Research Funding presented by the National Eye Institute, and entrepreneurship to help demystify some of the critical elements in positioning an emerging therapy into clinical trials and the marketplace. The group will also discuss the emerging retinal disease drug development landscape and give their personal views on the most exciting areas.

Below is a recording of the webinar.

Webinar #4 Panelists:

  • Rusty Kelley, Senior Vice President, Investments & Alliances, Foundation Fighting Blindness
    • Rusty Kelley, SVP, Investments & Alliances at Foundation Fighting Blindness, is responsible for external investment opportunities that strategically align with the Foundation’s therapeutic mission and its venture arm, the Retinal Degeneration Fund (RD Fund).  Prior to joining the Foundation, Rusty led the interdisciplinary and translational funds at the Burroughs Wellcome Fund (BWF).  Prior to BWF Rusty was the head of preclinical at Tengion, a tissue engineering biotech out of Boston Children’s and Wake Forest University.  Earlier in his career Rusty was under multiple divisions in clinical development at PPD.  Prior to PPD, Rusty was on the bench in the pharmaceutics and analytical chemistry divisions for AAIPharma.  Rusty received a BA in Chemistry from UNC Chapel Hill, a PhD in Pharmacology at LSU’s Health Sciences Center, was an American Heart Association postdoctoral fellowship in the School of Medicine at UNC Chapel Hill, and recently earned an MBA from Duke University. .
  • Paek Lee, Program Director, Small Business SBIT/STTR Program, NEI
    • Dr. Paek Lee is Program Director for NEI’s Small Business Innovation Research (SBIR) and Small Business Technology Transfer Research (STTR) portfolios and is also serving as NEI Research Resource Officer. Prior to joining NEI, Dr. Lee served as a Referral Officer and a Scientific Review Officer (SRO) at Center for Scientific Review (CSR) at NIH. As an SRO, he managed numerous NIH peer review panels including the Aging, Development, Auditory, Vision and Low Vision Technologies Small Business [ETTN-12] panel and the Molecular Neurogenetics [MNG] study section. Before joining CSR in 2003, he was an intramural researcher at the National Institute of Neurological Disorders and Stroke (NINDS), and he has a PhD in Neuroscience from Kent State University. His research experience was in the field of neuroscience, ranging from neurotrophic signaling to neuro-immune mechanisms underlying neurodegenerative conditions such as multiple sclerosis and diabetic neuropathy.
  • Deniz Dalkara, Scientific Director, Gamut Therapeutics
    • Deniz Dalkara is a tenured researcher in INSERM, France, and leads a team on gene therapies and animal models of neurodegenerative disease at the Vision Institute in Paris. She graduated from Middle East technical University with a B.S. degree in Biology in 2001. Afterwards, she obtained a masters degree in pharmacology and pharmacochemistry in Strasbourg, France where she later pursued a PhD degree in cellular and molecular aspects of biology. She was awarded the Biovalley PhD thesis for method of protein delivery developed during her graduate studies. Later on she conducted a postdoctoral fellowship in the laboratory of Ernst Babmerg at the Max Planck Institute of Biophysics before moving on to UC Berkeley to do a second post-doctoral training in 2007. At UC Berkeley, Dr. Dalkara applied viral engineering principals to enhance AAV vectors for their application in retinal degenerative diseases. Her work includes molecular evolution and engineering of viral gene delivery vehicles and their application to develop innovative gene therapeutic strategies to combat blinding diseases of the retina. For her work in this area, she received Euretina Science and Medicine Innovation award in 2013 and she was selected Innovator under 35 – France by MIT Technology Review in 2014. Dr. Dalkara received the Young Investigator award to start her group at the Vision Institute in Paris and has been carrying on her research activities in this research institute with a strong focus on translational research.

This webinar series is sponsored by