Event Details

Advance Beyond: Live Case Study Symposium, Welcoming Victoria Gray

We are thrilled to welcome Victoria Gray as keynote speaker to share her deeply personal and inspiring journey as the woman who became the world’s first gene-editing success story.

From Concept to Commercial, Gene Editing the Cure for Beta-Thalassemia and Sickle Cell Disease

Aldevron is taking a deeper dive into the linear path of gene editing as seen through the lens of curing beta thalassemia and sickle cell disease. The story tracks over 13 years where we welcome speakers covering the discovery of CRISPR, the identification of the dissection point on the gene, how the gene edited cell therapy product went from discovery to the clinic, and the regulatory approval pathway. We end with Victoria Gray, the woman who’s at the end of this success story—the first person to have their cells edited by CRISPR.

When
Thursday, June 12
8:00 am – 1:00 pm

Where
Sanctuary Event Center
Fargo, North Dakota
In-person and Virtual

Fee
No charge

Time Speaker / Event
7:15 Breakfast and networking
8:00 – 8:05 Welcome by Jen Meade, President
8:05 – 8:40 Fyodor Urnov, PhD
8:40 – 9:15 Dan Bauer, MD, PhD
9:15 – 9:50 Tirtha Chakraborty, PhD
9:50 – 10:15 Break
10:15 – 10:45 Tom Foti, VP / GM, Protein
10:45 – 11:20 Kimberly Benton, PhD
11:20 – 12:00 Victoria Gray, Keynote Speaker
12:00 – 12:30 Panel Discussion with Moderator Sadik Kassim, PhD
12:30 Box lunch to enjoy on site or to go

The timeline towards a cure

The timeline towards a cure for beta-thalassemia and sickle cell disease had specific and essential milestones to ensure the possibility became a reality.

2013
The story begins in the 1990s when scientists at Johns Hopkins and at the University of Cambridge first worked out how to engineer a protein to recognize a gene in human cells. A decade of work in industry and academia later, "genome editing 1.0" emerged and made its first forays into human clinical trials. Dr. Fyodor Urnov will tell the story of how fundamental insights into basic biological processes came together with industry innovation to advance the field to 2012 - the landmark year when Jennifer Doudna's and Emmanuelle Charpentier's Nobel-prize winning invention of gene editing using CRISPR which supercharged the field towards a cure.
2013-2015
Daniel Bauer, M.D., PhD., along with colleagues, inspired by human genetics, identified the BCL11A erythroid enhancer as a target for sickle cell disease gene editing. Disruption of key DNA sequences at BCL11A turns up the production of fetal hemoglobin, a potent therapeutic strategy. Dr. Bauer will share how he and his team pinpointed the specific region within the BCL11A gene’s erythroid enhancer that could be effectively edited by CRISPR-Cas9 to induce fetal hemoglobin.
2015-2018
Over the course of three years, while in multiple roles at CRISPR Therapeutics, Dr. Chakraborty led the team who drove the first gene-edited hematopoietic stem cell product from discovery and technology platform build to regulatory approval for beta-thalassemia and sickle cell disease. In lockstep with those efforts, Aldevron manufactured the RUO Cas9 and the world’s first GMP Cas9 for CRISPR therapeutics, which helped enable the path to the clinic.
2019
In July 2019, Victoria Gray became the first patient with sickle cell disease to receive a CRISPR-based cell therapy as a volunteer in the exa-cel clinical trial, sponsored by Vertex Pharmaceuticals and CRISPR Therapeutics. Barely four years later, the ensuing therapy, branded as Casgevy®, received approval from regulatory agencies in Europe, the United States, and the Middle East, ushering in a new era of CRISPR-based medicines.
2023
In December of 2023, the FDA approved the treatment for sickle cell disease in patients 12 years old and older. The treatment is the first CRISPR-based gene-editing therapy to be approved in the United States. Our speaker will provide the viewpoint from the regulatory perspective on how review and approval of this groundbreaking treatment occurred.
2025
Victoria Gray, the first patient with sickle cell disease to receive the CRISPR-based gene-editing therapy, will share her story of hope and inspiration.

Speakers

Victoria Gray
Picture Victoria Gray was the first sickle cell anemia patient in the world to be treated with CRISPR gene editing in 2019. After a lifetime of pain, treatments, and hospitalizations for sickle cell disease, she is now symptom-free, working as a patient advocate and international speaker to spread the word about CRISPR and rare diseases to clinicians, scientists, patients, and students. Mrs. Gray is a wife and mother of three children and lives in Forest, Mississippi.
Fyodor Urnov PhD
Picture

Fyodor Urnov is a Professor of Molecular Therapeutics at UC Berkeley and a Scientific Director at its Innovative Genomics Institute (IGI). He co-developed the toolbox of human genome and epigenome editing, co-named genome editing, and was on the team that advanced all of its first-in-human applications to the clinic. He also led the effort that identified the genome editing target for an approved medicine to treat sickle cell disease and beta-thalassemia.

A major goal for the field of genome editing and a key focus of Fyodor's work is expanding access to CRISPR therapies for genetic disease. As part of that effort Fyodor directs the Danaher-IGI Beacon for CRISPR Cures - a first-in-class academia-industry partnership developing and advancing to the clinic scalable CRISPR-based approaches to treat diseases of the immune system.

Daniel E. Bauer, MD PhD
Picture

Daniel Bauer is a physician-scientist whose research focuses on functional genomics to dissect determinants of blood disorders and develop innovative therapies.

He discovered the gene editing approach targeting the BCL11A erythroid enhancer that was developed as exa-cel, now approved for sickle cell disease and β-thalassemia treatment. He has described mechanisms of fetal hemoglobin (HbF) repression, demonstrated how human genetic diversity alters therapeutic gene editing outcomes, and uncovered how the hematopoietic stem cell state constrains the efficiency and genotoxicity of nuclease, base and prime editing.

He has sponsored investigator-initiated clinical trials of therapeutic gene editing. His clinical work focuses on non-malignant hematology.

He is Donald S. Fredrickson, MD Associate Professor of Pediatrics at Harvard Medical School and Director of the Gene Therapy Program at Boston Children’s Hospital.

Tirtha Chakraborty, PhD
Picture

Dr. Tirtha Chakraborty specializes in Genetic Medicines. In his most recent position, he was the Chief Scientific Officer and Head of Technical Operations at Vor Bio, a clinical stage company, focused on AML treatment using hematopoietic transplant and immuno-therapy. At Vor, he oversaw both Research as well as Process and Analytical Development and GMP manufacturing. His team was responsible for the early Research and Development of trem-cel, a CD33-null allogeneic transplant and VCAR33, a CD33 directed CAR-T therapy. Both cellular medicines were in clinical trials.

Before joining Vor, Dr. Chakraborty was the VP of Cell Therapy Research at Sana Biotechnology. Prior to Sana, he was the Head of Hematology at CRISPR Therapeutics, leading the platform development and pre-clinical work for regulatory filing of the first ever genome-edited transplant, Casgevy (exa-cel), for Beta-thalassemia and Sickle Cell Disease.

Prior to that, Dr. Chakraborty oversaw the therapeutic mRNA technology platform development at Moderna Therapeutics, paving the foundation for the mRNA COVID-19 vaccines. In his academic life, he was trained as a molecular geneticist and investigated the role of post-transcriptional gene regulation in lymphocyte development and lymphomagenesis using genetic engineering.

Tom Foti
Picture

In 2009, Tom Foti co-founded Aldevron’s Protein Business Unit and currently serves in the role of Vice President / General Manager. Aldevron’s Protein Business Unit offers development and manufacturing services to the research, diagnostic and therapeutic markets and is based in Madison, WI.

Tom has been instrumental in developing and launching the CRISPR product line at Aldevron. From off-the-shelf products to custom GMP manufacturing, he and his team have helped clients advance their programs from concept to the clinic. His guidance and foresight have kept Aldevron in a leadership position in the competitive gene and cell therapy markets.

Tom has more than 30 years of biotechnology experience and prior to joining Aldevron, he served in several roles in Merck KGaA’s Bioscience Division (2000-2008), most recently as the Director of its Global Custom Services Business. He started his career in 1992, with Novagen, Inc. serving in scientific, 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.

Sadik H. Kassim
Picture

Sadik H. Kassim is a highly-regarded scientist and executive with extensive experience in biotechnology with a specific focus on cell and gene therapy bioprocessing and translational research. Currently, he is Chief Technology Officer at Danaher with a focus on genomic medicines. He previously was Chief Technology Officer at Vor Bio where he built the technical operations team responsible for process development, analytical development, supply chain, and manufacturing support of a CRISPR gene-edited HSPC product and oversaw the company’s CAR-T efforts. Prior to Vor, Sadik was Executive Director at Kite Pharma and led development of manufacturing processes for autologous CAR-T and TCR-based cell therapies. As the Chief Scientific Officer at Mustang Bio, Sadik managed the foundational build-out of the company’s preclinical and manufacturing activities.

Earlier in his career, Sadik was Head of Early Analytical Development for Novartis’ Cell and Gene Therapies Unit and worked on research teams at the National Cancer Institute with Dr. Steven Rosenberg, the University of Pennsylvania Gene Therapy Program with Dr. Jim Wilson, and Johnson and Johnson’s Immunology Discovery group. Sadik and his teams have contributed to the successful BLA and MAA applications for three of the commercially available CAR-T therapies: Kymriah®, Yescarta®, and Tecartus®. Sadik holds a B.S. degree in cell and molecular biology from Tulane University and earned his Ph.D. in Microbiology and Immunology from Louisiana State University.

Kimberly Benton, PhD
Picture Dr Kimberly Benton is Master Principal and Head of Regulatory at Dark Horse Consulting Group. At DHC, she provides regulatory expertise and strategic perspective to a broad range of clients in the cell and gene therapy field. Prior to joining DHC, Kim served for over 22 years at the US Food and Drug Administration, Center for Biologics Evaluation and Research (CBER). Her last position at CBER was She served as Associate Director for Regulatory Management in the Office of Tissues and Advanced Therapies (now known as the Office of Therapeutic Products). In this role, she directed the regulatory review program for the broad portfolio of products under OTAT’s purview: human tissues, cellular therapies, gene therapies, therapeutic vaccines, xenotransplantation products, and purified and recombinant versions of therapeutic proteins for hematology. For the majority of her career at FDA, she directed chemistry, manufacturing, and controls review, serving in prior positions as Deputy Director of the Division of Cellular and Gene Therapies in the Office of Cellular, Tissue, and Gene Therapies, and Chief of the Cell Therapies Branch.
RSVP Today!
rsvp
#878686
region na1
portalId 1769030
formId 990f6402-9590-4e31-af80-38053a65a098
target services-hubspot-form