The Cell and Gene Therapy (CGT) Science Series is a quarterly seminar series focused on scientific topics related to cell and gene therapy products. The CGT Science Series is intended to foster scientific exchange between the Biotechnology Innovation Organization (BIO), the American Society of Cell & Gene Therapy (ASGCT), and Center for Biologics Evaluation and Research (CBER) review staff on a variety of topics that span the CGT product lifecycle. The seminars are planned as 60-minute virtual webinars featuring a speaker from one of the three organizations. The CGT Science Series will enable a deep dive into a specific technical and/or scientific area. Topics in the series may include but are not limited to, nonclinical, CMC, clinical, or post-market phases of development related to CGT product lifecycle.

Moderator:
– Anne-Virginie Eggimann, Tessera

Opening Remarks:
– Cartier Esham, Chief Scientific Officer, Biotechnology Innovation Organization (BIO)

Speaker:
– Jing Liao, Director, Vector Development and Operations, Alexion Pharmaceuticals

Cell + Gene Therapy Science Series

An ASGCT collaboration with BIO and FDA

The Cell and Gene Therapy (CGT) Science Series is a quarterly seminar series focused on scientific topics related to cell and gene therapy products. The CGT Science Series is intended to foster scientific exchange between the Biotechnology Innovation Organization (BIO), American Society of Gene & Cell Therapy (ASGCT), and FDA's Center for Biologics Evaluation and Research (CBER) review staff on a variety of topics that span the CGT product lifecycle. The seminars are planned as 60-minute virtual webinars featuring a speaker from one of the three organizations. The CGT Science Series will enable a deep dive into a specific technical and/or scientific area.  Topics in the series may include, but are not limited to, nonclinical, CMC, clinical, or post-market phases of development related to CGT product lifecycle. 

Short and Long Read Sequencing for AAV Characterization

The inaugural CGT Science Series seminar  focused on NGS characterization of AAV and was presented by Dr. Lauriel Earley. 

Dr. Earley is a Senior Scientist with Shape Therapeutics who has been studying the basic biology of adeno-associated virus for over decade. During her graduate studies at Oregon Health & Science University, she examined the role of assembly-activating protein under the mentorship of Dr. Hiroyuki Nakai. After receiving her PhD, she joined Dr. Jude Samulski's laboratory at the University of North Carolina, Chapel Hill and took on a project to characterize the intrinsic promoter ability of the inverted terminal repeat sequences from various AAV serotypes. Dr. Earley joined Shape Therapeutics in 2019 and is currently leading their vector platform development team. In addition, Dr. Earley has been an active member of ASGCT since 2012 and is currently serving on the Patient Outreach Committee.

This is the first meeting in a series of conferences cohosted by the Biotechnology Innovation Organization (BIO) and Solve M.E. that are designed to foster a private-public engagement and create an R&D roadmap for diagnostics and treatments for Long COVID and Post-infection Diseases.

Objective: Convene stakeholders to advance R&D to diagnose and treat Long COVID and post-infection diseases to:

--Increase awareness among drug developers to the unmet need and business opportunity in these indications

--Establish a knowledge base of emerging research in Long COVID and existing body of data in other post-infection diseases (such as dysautonomia, myalgic encephalomyelitis/ chronic fatigue syndrome, mast cell activation, and others).

--Highlight potential biological targets for therapeutic and preventative approaches

--Engage experts and regulators to inform design of clinical studies

--Establish a pre-competitive collaborative network

Recorded on February 21, 2023. Part 1 of 3.

This is the first meeting in a series of conferences cohosted by the Biotechnology Innovation Organization (BIO) and Solve M.E. that are designed to foster a private-public engagement and create an R&D roadmap for diagnostics and treatments for Long COVID and Post-infection Diseases.

Objective: Convene stakeholders to advance R&D to diagnose and treat Long COVID and post-infection diseases to:

--Increase awareness among drug developers to the unmet need and business opportunity in these indications

--Establish a knowledge base of emerging research in Long COVID and existing body of data in other post-infection diseases (such as dysautonomia, myalgic encephalomyelitis/ chronic fatigue syndrome, mast cell activation, and others).

--Highlight potential biological targets for therapeutic and preventative approaches

--Engage experts and regulators to inform design of clinical studies

--Establish a pre-competitive collaborative network

Recorded on February 21, 2023. Part 2 of 3.

This is the first meeting in a series of conferences cohosted by the Biotechnology Innovation Organization (BIO) and Solve M.E. that are designed to foster a private-public engagement and create an R&D roadmap for diagnostics and treatments for Long COVID and Post-infection Diseases.

Objective: Convene stakeholders to advance R&D to diagnose and treat Long COVID and post-infection diseases to:

--Increase awareness among drug developers to the unmet need and business opportunity in these indications

--Establish a knowledge base of emerging research in Long COVID and existing body of data in other post-infection diseases (such as dysautonomia, myalgic encephalomyelitis/ chronic fatigue syndrome, mast cell activation, and others).

--Highlight potential biological targets for therapeutic and preventative approaches

--Engage experts and regulators to inform design of clinical studies

--Establish a pre-competitive collaborative network

Recorded on February 21, 2023. Part 3 of 3.

Innovation flourishes when science and consumer values are aligned and complement one another. Regulatory approaches towards innovative products cannot exist in isolation. They should be supported by credible transparency measures. A proactive approach to transparency stands to build trust and foster an inclusive environment to address our most pressing societal, nutritional, and environmental concerns. This webinar will explore the meaningful – and sometimes unexpected- actions and partnerships that are growing trust in the innovation ecosystem and delivering access to innovations that benefit people and the planet. 
 

New algorithms and optical system improvements are expanding the frontier of cell imaging quality. Artificial intelligence techniques enable better visualization of cellular mechanisms in a wide number of applications and across multiple scientific disciplines, including advancing neurological research. View this Biotechnology Innovation Organization (BIO) webinar as it highlights how AI and imaging are being used to improve our understanding of complex and notoriously intractable disorders such as Alzheimer’s, Dementia, and related diseases. Learn from the following imaging and therapeutic development experts as they answer audience questions during the session.  

The Biotechnology Innovation Organization (BIO) and BioFlorida present "The Transformative Potential of Genomic Medicine 2.0." Genomic technologies create powerful tools that have the potential to diagnose, treat, and permanently subdue many human diseases. Technologies such as gene therapy, gene editing, and CRISPR are just some of the newest terms as researchers and clinicians work on new approaches to develop lifesaving, disease-altering treatments. This session provides in-depth discussion regarding how treatment is changing in rare diseases and the sickle cell community, and how public and private payers are approaching coverage of these crucial treatments. The session opens with statements from two legislators, then the panel features two patients and an expert in alternative payment models to provide information and answer questions from policymakers.  

Representative Sam Garrison
Florida House of Representatives
House Health and Human Services Committee Republican Majority Whip

Senator Bobby Powell
Florida State Senate
Legislative Black Caucus Chair