Resources

Novel expertise for novel therapies

Overviews

Precision ADVANCE Overview

Precision ADVANCE Clinical Services Overview

Precision ADVANCE Commercial Services Overview

Precision ADVANCE Manufacturing Services Overview

Briefs

Precision ADVANCE Events & Expertise

Gene Therapy for Rare Disorders Panel Summary

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

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

ASGCT Panel Part 1: Cell Therapy

ASGCT Panel Part 2: Gene Therapy

Meeting on the Med Panel

Precision ADVANCE Company Presentation: Meeting on the Med

Endpoints Webinar: The Great Debate: Internal vs External Manufacturing for Advanced Therapies

Gene Therapy for Rare Disorders Panel

BioProcess Interview: Tackling the Cell & Gene Manufacturing Talent Crunch

Precision ADVANCE Overview

Precision ADVANCE Clinical Services Overview

Precision ADVANCE Commercial Services Overview

Precision ADVANCE Manufacturing Services Overview

Precision ADVANCE Events & Expertise

Gene Therapy for Rare Disorders Panel Summary

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

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.

ASGCT Panel Part 1: Cell Therapy

ASGCT Panel Part 2: Gene Therapy

Meeting on the Med Panel

Precision ADVANCE Company Presentation: Meeting on the Med

Endpoints Webinar: The Great Debate: Internal vs External Manufacturing for Advanced Therapies

Gene Therapy for Rare Disorders Panel

BioProcess Interview: Tackling the Cell & Gene Manufacturing Talent Crunch

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 Events & Expertise

Gene Therapy for Rare Disorders Panel Summary

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