Cell and Gene Therapy Resources: Case Studies, White Papers, and More
Novel expertise for novel therapies with Precision ADVANCE’s Cell and Gene Therapy Resources
Overviews
Precision ADVANCE Overview
Precision ADVANCE Clinical Services Overview
Precision ADVANCE Commercial Services Overview
Precision ADVANCE Manufacturing Services Overview
Briefs
Precision ADVANCE Expertise
WODC Panel Summary: Pricing & Reimbursement for Rare Diseases in the United States
WODC Panel Summary: Outlook on European Market Access Strategies for Rare Diseases
The Future of Reimbursement for Cell & Gene Therapies
Precision ADVANCE at ISPOR 2021
Case Studies
Building the first-of-its kind gene therapy facility, commercializing one of the first gene therapies and industrializing the largest gene therapy footprint in the world
Navigating the CAR T-cell Therapy Landscape to Develop Market Access and Distribution Strategies
Process and Communication: Navigating Logistical Complexity in Matched Cellular Therapy Trials
Mining Stakeholder Insights on Access to Gene Therapies for Hemophilia
The Value of Gene Therapy in Spinal Muscular Atrophy
Ensuring Provider Education And Readiness For Using A Novel CAR-T
White Papers
Unleashing the Power of Precision Medicine: Radioligand Therapy
Scaling Hope: The Growth of Allogeneic Cell Therapy Sector
Cell and Gene Therapies: How High Can Prices Go?
mRNA Innovation Is Revolutionizing Disease Prevention
Moving Clinical Development Forward in 2023
The Future is Now: Cell and Gene Therapy Innovation, Challenges, and Perspectives
The Price is Right: Cell and Gene Therapy Approvals and Market Access in 2023
State of the Industry
“Preventative” Gene Therapies: Perspectives on Payer Access Challenges and Solutions
Gene Editing Breakthroughs: A New Hope for Patients
Moving CGTx Clinical Development Forward in 2023
Market Trends and Expectations for Advanced Therapies in 2023
What’s Next for Cell and Gene Therapies in 2023 and Beyond
State of the Union for Advanced Medicines
Considerations for the Clinical Development of Cell & Gene Therapies (Part 1: Cell Therapies)
Considerations for the Clinical Development of Cell & Gene Therapies (Part 2: Gene Therapies)
Innovation: Shortening the Path to Commercialization in Advanced Therapies
The Great Debate: Internal vs. External Manufacturing for Advanced Therapies
The Future of Reimbursement for Cell and Gene Therapies
Gene Therapies in Hemophilia
Considerations for Early-Stage Gene Therapy Start-ups: From Clinical Development to Manufacturing to Commercialization
Delivering on the Promise of Cell Therapy: Challenges and Trends
Optimizing Successful Development of Viral Vector Gene Therapies, Gene Therapy Trials, and Companion Diagnostics
Perspectives on Gene Therapy: Defining and Demonstrating Value to Payers
Advantages of Using POD Cleanroom Technology
What to Consider When Conducting a “Make vs. Buy” Analysis for a Cell & Gene Therapy Facility
Publications
Costs and health resource use in patients with X-linked myotubular myopathy: insights from U.S. commercial claims. Journal of Managed Care & Specialty Pharmacy. 2021.
An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). Journal of Market Access & Health Policy. 2020.
Economic Burden of X-Linked Myotubular Myopathy (XLMTM) By Ventilation Status. ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Healthcare resource use and expenditures in patients with X-linked myotubular myopathy (XLMTM). ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Health resource use in patients with x-linked myotubular myopathy (XLMTM): Data from the RECENSUS Study. ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays. AAPS Journal. 2020.
Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients. Journal of Market Access & Health Policy. 2019.
Immunogenicity of Chimeric Antigen Receptor T-Cell Therapeutics. Springer Nature AG. 2019.
Economic assessment of novel gene and cellular therapies: beyond traditional modeling. J Clin Pathways. 2018.
Videos
The Future of Cell Therapy Development and Delivery (Endpoints Webinar)
The Price is Right: Approvals & Market Access in 2023 (CGTx Day, 2023)
Moving Clinical Development Forward in 2023 (CGTx Day, 2023)
The Future is Now: CGTx innovation, challenges, and perspectives (CGTx Day, 2023)
State of the Industry Briefing by Tim Hunt (CGTx Day, 2023)
The Hitchhiker’s Guide to CGTx in 2023 (Cell & Gene Webinar)
State of the Union for Advanced Medicines (Endpoints Webinar)
Overcoming Manufacturing Challenges for Advanced Medicines (Endpoints Webinar)
Gene Therapy Roundup: What to Know Now from the Lab to the FDA (Endpoints Webinar)
What’s Next for Cell and Gene Therapies in 2023 and Beyond (Meeting on the Mesa Conference, 2022)
A Crisis in Rare Disease (Endpoints Webinar)
The Next Normal: Predictions for the Future of Advanced Medicines (Meeting on the Med Conference, 2022)
Public Markets’ Impact on Manufacturing Advanced Therapies (Endpoints Webinar)
Planning for the Commercialization of Advanced Therapies (Endpoints Webinar)
CQV Strategies that Increase Speed-to-Market for Gene Therapy (Endpoints Webinar)
State of the Union for Advanced Medicines (Endpoints Webinar)
CQV Strategies to Optimize Cell Therapy Manufacturing (Endpoints Webinar)
Scaling Up a Gene Therapy Manufacturing Facility (Endpoints Webinar)
Skills: The Global Gold Rush (Advanced Therapies Integrates Panel)
Pricing Strategies for Advanced Therapies (Endpoints Webinar)
The Future of Cell Therapy: Auto & Allo (CAR-TCR Panel)
Strategies for Building a Cell Therapy Manufacturing Facility (Endpoints Webinar)
Development of a Neutralizing Antibody Assay (Gene Therapy Immunogenicity Presentation)
Essential Strategies for Gene Therapy (Endpoints Webinar)
The Future of Reimbursement for Cell and Gene Therapies (Precision Webinar)
Considerations for the Clinical Development of a Cell Therapy (ASGCT Panel Pt.1)
Considerations for the Clinical Development of a Gene Therapy (ASGCT Panel Pt.2)
Shortening the Path to Commercialization in Advanced Therapies (Meeting on the Med Panel)
The Great Debate: Internal vs. External Manufacturing for Advanced Therapies (Endpoints Webinar)
Considerations for Early-Stage Gene Therapy Start-Ups (GTRD Panel)
Tackling the Cell & Gene Manufacturing Talent Crunch (BioProcess Interview)
Precision ADVANCE Overview
Precision ADVANCE Clinical Services Overview
Precision ADVANCE Commercial Services Overview
Precision ADVANCE Manufacturing Services Overview
Precision ADVANCE Expertise
WODC Panel Summary: Pricing & Reimbursement for Rare Diseases in the United States
WODC Panel Summary: Outlook on European Market Access Strategies for Rare Diseases
The Future of Reimbursement for Cell & Gene Therapies
Precision ADVANCE at ISPOR 2021
Building the first-of-its kind gene therapy facility, commercializing one of the first gene therapies and industrializing the largest gene therapy footprint in the world
Navigating the CAR T-cell Therapy Landscape to Develop Market Access and Distribution Strategies
Process and Communication: Navigating Logistical Complexity in Matched Cellular Therapy Trials
Mining Stakeholder Insights on Access to Gene Therapies for Hemophilia
The Value of Gene Therapy in Spinal Muscular Atrophy
Ensuring Provider Education And Readiness For Using A Novel CAR-T
Unleashing the Power of Precision Medicine: Radioligand Therapy
Scaling Hope: The Growth of Allogeneic Cell Therapy Sector
Cell and Gene Therapies: How High Can Prices Go?
mRNA Innovation Is Revolutionizing Disease Prevention
Moving Clinical Development Forward in 2023
The Future is Now: Cell and Gene Therapy Innovation, Challenges, and Perspectives
The Price is Right: Cell and Gene Therapy Approvals and Market Access in 2023
State of the Industry
“Preventative” Gene Therapies: Perspectives on Payer Access Challenges and Solutions
Gene Editing Breakthroughs: A New Hope for Patients
Moving CGTx Clinical Development Forward in 2023
Market Trends and Expectations for Advanced Therapies in 2023
What’s Next for Cell and Gene Therapies in 2023 and Beyond
State of the Union for Advanced Medicines
Considerations for the Clinical Development of Cell & Gene Therapies (Part 1: Cell Therapies)
Considerations for the Clinical Development of Cell & Gene Therapies (Part 2: Gene Therapies)
Innovation: Shortening the Path to Commercialization in Advanced Therapies
The Great Debate: Internal vs. External Manufacturing for Advanced Therapies
The Future of Reimbursement for Cell and Gene Therapies
Gene Therapies in Hemophilia
Considerations for Early-Stage Gene Therapy Start-ups: From Clinical Development to Manufacturing to Commercialization
Delivering on the Promise of Cell Therapy: Challenges and Trends
Optimizing Successful Development of Viral Vector Gene Therapies, Gene Therapy Trials, and Companion Diagnostics
Perspectives on Gene Therapy: Defining and Demonstrating Value to Payers
Advantages of Using POD Cleanroom Technology
What to Consider When Conducting a “Make vs. Buy” Analysis for a Cell & Gene Therapy Facility
Costs and health resource use in patients with X-linked myotubular myopathy: insights from U.S. commercial claims. Journal of Managed Care & Specialty Pharmacy. 2021.
An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). Journal of Market Access & Health Policy. 2020.
Economic Burden of X-Linked Myotubular Myopathy (XLMTM) By Ventilation Status. ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Healthcare resource use and expenditures in patients with X-linked myotubular myopathy (XLMTM). ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Health resource use in patients with x-linked myotubular myopathy (XLMTM): Data from the RECENSUS Study. ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays. AAPS Journal. 2020.
Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients. Journal of Market Access & Health Policy. 2019.
Immunogenicity of Chimeric Antigen Receptor T‑Cell Therapeutics. Springer Nature AG. 2019.
Economic assessment of novel gene and cellular therapies: beyond traditional modeling. J Clin Pathways. 2018.
The Future of Cell Therapy Development and Delivery (Endpoints Webinar)
The Price is Right: Approvals & Market Access in 2023 (CGTx Day, 2023)
Moving Clinical Development Forward in 2023 (CGTx Day, 2023)
The Future is Now: CGTx innovation, challenges, and perspectives (CGTx Day, 2023)
State of the Industry Briefing by Tim Hunt (CGTx Day, 2023)
The Hitchhiker’s Guide to CGTx in 2023 (Cell & Gene Webinar)
State of the Union for Advanced Medicines (Endpoints Webinar)
Overcoming Manufacturing Challenges for Advanced Medicines (Endpoints Webinar)
Gene Therapy Roundup: What to Know Now from the Lab to the FDA (Endpoints Webinar)
What’s Next for Cell and Gene Therapies in 2023 and Beyond (Meeting on the Mesa Conference, 2022)
A Crisis in Rare Disease (Endpoints Webinar)
The Next Normal: Predictions for the Future of Advanced Medicines (Meeting on the Med Conference, 2022)
Public Markets’ Impact on Manufacturing Advanced Therapies (Endpoints Webinar)
Planning for the Commercialization of Advanced Therapies (Endpoints Webinar)
CQV Strategies that Increase Speed-to-Market for Gene Therapy (Endpoints Webinar)
State of the Union for Advanced Medicines (Endpoints Webinar)
CQV Strategies to Optimize Cell Therapy Manufacturing (Endpoints Webinar)
Scaling Up a Gene Therapy Manufacturing Facility (Endpoints Webinar)
Skills: The Global Gold Rush (Advanced Therapies Integrates Panel)
Pricing Strategies for Advanced Therapies (Endpoints Webinar)
The Future of Cell Therapy: Auto & Allo (CAR-TCR Panel)
Strategies for Building a Cell Therapy Manufacturing Facility (Endpoints Webinar)
Development of a Neutralizing Antibody Assay (Gene Therapy Immunogenicity Presentation)
Essential Strategies for Gene Therapy (Endpoints Webinar)
The Future of Reimbursement for Cell and Gene Therapies (Precision Webinar)
Considerations for the Clinical Development of a Cell Therapy (ASGCT Panel Pt.1)
Considerations for the Clinical Development of a Gene Therapy (ASGCT Panel Pt.2)
Shortening the Path to Commercialization in Advanced Therapies (Meeting on the Med Panel)
The Great Debate: Internal vs. External Manufacturing for Advanced Therapies (Endpoints Webinar)
Considerations for Early-Stage Gene Therapy Start-Ups (GTRD Panel)
Tackling the Cell & Gene Manufacturing Talent Crunch (BioProcess Interview)
Resources
Novel expertise for novel therapies
Why Precision ADVANCE?
Precision ADVANCE Overview
Precision ADVANCE Clinical Services Overview
Precision ADVANCE Commercial Services Overview
Precision ADVANCE Manufacturing Services Overview
Briefs
Precision ADVANCE Expertise
Precision ADVANCE at ISPOR 2021
Precision ADVANCE at ISPOR 2021
Precision ADVANCE at ISPOR 2021
Precision ADVANCE at ISPOR 2021
Case studies
Building the first-of-its kind gene therapy facility, commercializing one of the first gene therapies and industrializing the largest gene therapy footprint in the world
Navigating the CAR T-cell Therapy Landscape to Develop Market Access and Distribution Strategies
Process and Communication: Navigating Logistical Complexity in Matched Cellular Therapy Trials
Mining Stakeholder Insights on Access to Gene Therapies for Hemophilia
The Value of Gene Therapy in Spinal Muscular Atrophy
Ensuring Provider Education And Readiness For Using A Novel CAR-T
White papers
Unleashing the Power of Precision Medicine: Radioligand Therapy
Scaling Hope: The Growth of Allogeneic Cell Therapy Sector
Cell and Gene Therapies: How High Can Prices Go?
mRNA Innovation Is Revolutionizing Disease Prevention
Moving Clinical Development Forward in 2023
The Future is Now: Cell and Gene Therapy Innovation, Challenges, and Perspectives
The Price is Right: Cell and Gene Therapy Approvals and Market Access in 2023
State of the Industry
“Preventative” Gene Therapies: Perspectives on Payer Access Challenges and Solutions
Gene Editing Breakthroughs: A New Hope for Patients
Moving CGTx Clinical Development Forward in 2023
Market Trends and Expectations for Advanced Therapies in 2023
What’s Next for Cell and Gene Therapies in 2023 and Beyond
State of the Union for Advanced Medicines
Considerations for the Clinical Development of Cell & Gene Therapies (Part 1: Cell Therapies)
Considerations for the Clinical Development of Cell & Gene Therapies (Part 2: Gene Therapies)
Innovation: Shortening the Path to Commercialization in Advanced Therapies
The Great Debate: Internal vs. External Manufacturing for Advanced Therapies
The Future of Reimbursement for Cell and Gene Therapies
Gene Therapies in Hemophilia
Considerations for Early-Stage Gene Therapy Start-ups: From Clinical Development to Manufacturing to Commercialization
Delivering on the Promise of Cell Therapy: Challenges and Trends
Optimizing Successful Development of Viral Vector Gene Therapies, Gene Therapy Trials, and Companion Diagnostics
Perspectives on Gene Therapy: Defining and Demonstrating Value to Payers
Advantages of Using POD Cleanroom Technolog
What to Consider When Conducting a “Make vs. Buy” Analysis for a Cell & Gene Therapy Facility
Developing Advanced Therapies: Considerations for Internal Versus External Manufacturing
Publications
Costs and health resource use in patients with X-linked myotubular myopathy: insights from U.S. commercial claims. Journal of Managed Care & Specialty Pharmacy. 2021.
An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). Journal of Market Access & Health Policy. 2020.
Economic Burden of X-Linked Myotubular Myopathy (XLMTM) By Ventilation Status. ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Healthcare resource use and expenditures in patients with X-linked myotubular myopathy (XLMTM). ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Health resource use in patients with x-linked myotubular myopathy (XLMTM): Data from the RECENSUS Study. ISPOR 25th Annual International Meeting. Orlando FL. 2020.
Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays. AAPS Journal. 2020.
Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients. Journal of Market Access & Health Policy. 2019.
Immunogenicity of Chimeric Antigen Receptor T‑Cell Therapeutics. Springer Nature AG. 2019.
Economic assessment of novel gene and cellular therapies: beyond traditional modeling. J Clin Pathways. 2018.
Videos
The Future of Cell Therapy Development and Delivery (Endpoints Webinar)
The Price is Right: Approvals & Market Access in 2023 (CGTx Day, 2023)
Moving Clinical Development Forward in 2023 (CGTx Day, 2023)
The Future is Now: CGTx innovation, challenges, and perspectives (CGTx Day, 2023)
State of the Industry Briefing by Tim Hunt (CGTx Day, 2023)
The Hitchhiker’s Guide to CGTx in 2023 (Cell & Gene Webinar)
State of the Union for Advanced Medicines (Endpoints Webinar)
Overcoming Manufacturing Challenges for Advanced Medicines (Endpoints Webinar)
Gene Therapy Roundup: What to Know Now from the Lab to the FDA (Endpoints Webinar)
CQV Strategies that Increase Speed-to-Market for Gene Therapy (Endpoints Webinar)
A Crisis in Rare Disease (Endpoints Webinar)
The Next Normal: Predictions for the Future of Advanced Medicines (Meeting on the Med Conference, 2022)
The Next Normal: Predictions for the Future of Advanced Medicines (Meeting on the Med Conference, 2022)
CQV Strategies that Increase Speed-to-Market for Gene Therapy (Endpoints Webinar)
CQV Strategies that Increase Speed-to-Market for Gene Therapy (Endpoints Webinar)
State of the Union for Advanced Medicines (Endpoints Webinar)
CQV Strategies to Optimize Cell Therapy Manufacturing (Endpoints Webinar)
Scaling Up a Gene Therapy Manufacturing Facility (Endpoints Webinar)
Skills: The Global Gold Rush (Advanced Therapies Integrates Panel)
Pricing Strategies for Advanced Therapies (Endpoints Webinar)
The Future of Cell Therapy: Auto & Allo (CAR-TCR Panel)
Strategies for Building a Cell Therapy Manufacturing Facility (Endpoints Webinar)
Development of a Neutralizing Antibody Assay (Gene Therapy Immunogenicity Presentation)
Essential Strategies for Gene Therapy (Endpoints Webinar)
The Future of Reimbursement for Cell and Gene Therapies (Precision Webinar)
Considerations for the Clinical Development of a Cell Therapy (ASGCT Panel Pt.1)
Considerations for the Clinical Development of a Gene Therapy (ASGCT Panel Pt.2)
Shortening the Path to Commercialization in Advanced Therapies (Meeting on the Med Panel)
The Great Debate: Internal vs. External Manufacturing for Advanced Therapies (Endpoints Webinar)
Considerations for Early-Stage Gene Therapy Start-Ups (GTRD Panel)
Tackling the Cell & Gene Manufacturing Talent Crunch (BioProcess Interview)
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