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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

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

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

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

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

Developing Advanced Therapies: Considerations for Internal Versus External Manufacturing

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.

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)