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Return of Pipeline 2026 – Cell and Gene Therapies

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In Part 2 of the 2026 pipeline series, host Carolyn Liptak welcomes Dr. Amanda Frick, Senior Clinical Manager, Strategic Clinical Intelligence at Vizient, to explore the advanced therapies pipeline: cell therapies, gene therapies, tissue-engineered products, and combination advanced therapy products.

The discussion explores major pipeline trends, six leading products to watch, and the growing innovation expected to shape clinical practice in 2026.

 

Guest speakers: 

Amanda Frick, PharmD, BCPS

Senior Clinical Manager, Strategic Clinical Intelligence

Spend Management

Vizient


Host:

Carolyn Liptak, MBA, BS Pharm

Pharmacy Executive Director

Center for Pharmacy Practice Excellence (CPPE)

Vizient 

 

Show Notes:

[00:05] — Introduction

  • Announcer opens the episode.
  • Host Carolyn Liptak introduces the focus on advanced therapies:
    • cell & gene therapies, tissue-engineered products, and combination products.
  • Guest: Dr. Amanda Frick, Senior Clinical Manager, Strategic Clinical Intelligence at Vizient.

    • [01:07] — Defining Advanced Therapies

    • FDA groups cell and gene therapies within advanced therapies.
    • Total FDA-approved advanced therapies: 46.
    • Amanda monitors 29 drug-like therapies within that group.

      • [02:01] — Pipeline Size and Approval Activity

      • S. pipeline: 264 agents in development.
      • About 10 agents approach FDA decision annually.
      • Actual approvals: 5–7 per year on average.

        • [02:56] — Big-Picture Trends in Cell & Gene Therapy

        Oncology dominates

        • 40–50% of all CGTs in development.
        • Expanding into autoimmune, neurology, and earlier-phase therapies for diabetes, angina, osteoarthritis.

          • Movement toward allogeneic ("off-the-shelf") therapies

          • Designed to overcome limits of autologous cell manufacturing.
          • Reduces wait time and manufacturing failures.

            • Resurgence of therapeutic vaccines

            • Currently 3 approved (Sipuleucel-T, Talimogene, Papzimeos
            • ).
            • 20+ vaccines in the pipeline, largely targeted to cancer.
            • CE program coming Jan 29.

              • [06:13] — Therapy #1:  Tabelecleucel or Tab-cel (Allogeneic EBV-Specific T-Cell Therapy)

              • First allogeneic T-cell therapy expected in the U.S.
              • For EBV-positive post-transplant lymphoproliferative disorder (PTLD).
              • “Off-the-shelf” and donor-derived.

                • [07:07] — Clinical Need & Outcomes

                • Currentstandard of care: rituximab.
                • After relapse, survival <1 month (HSCT) or <4 months (solid organ).
                • Phase 3 data: 90% one-year survival.

                  • Regulatory Status

                  • Prior FDA CRL due to manufacturing (third-party issues).
                  • Expected approval: January 10, 2026.

                    • [09:00] — Therapy #2:  Kresladi  (Autologous Gene Therapy for LAD-I)

                    • Treats severe leukocyte adhesion deficiency (LAD-I), typically diagnosed in infancy.
                    • Could eliminate need for stem cell transplant (often limited by lack of matched donors).

                      • [11:39] — Clinical Outcomes

                      • Phase 1/2 data (n=9):
                        • 100% survival at one year
                        • Major reduction in hospitalizations
                        • Discontinuation of prophylactic antibiotics

                          • Regulatory Status

                          • Multiple delays due to CMC (chemistry, manufacturing, controls) questions.
                          • Resubmitted Oct 2025; anticipated approval March 28, 2026.

                            • [13:27] — Therapy #3: ORCA-T (Enhanced Allogeneic Stem Cell Transplant)

                            • Allogeneic hematopoietic stem cells + regulatory and conventional T cells (Tregs + Tcons).
                            • Designed to improve:
                              • Graft-versus-tumor effect
                              • Graft-versus-infection protection
                              • Reduction in graft-versus-host disease (GVHD)

                                • [14:31] — Clinical Significance

                                • Could markedly improve quality of life after transplant.

                                  • Regulatory Status

                                  • Anticipated approval: April 6, 2026.

                                    • [15:01] — Therapy #4: Vusolimogene oderparepvec (RP1) (RP1 — Oncolytic HSV-1 Virus)

                                    • Off-the-shelf genetically modified HSV-1 oncolytic virus.
                                    • For advanced melanoma after prior PD-1 inhibitor therapy.

                                      • [16:17] — Why It Matters

                                      • Currently, poised to compete with Amtagvi (TIL therapy).
                                      • TIL therapy is autologous and slow to manufacture; RP1 is ready-to-use and combines with nivolumab (Opdivo).

                                        • Regulatory Status

                                        • PDUFA: April 10, 2026.

                                          • [16:34] — Therapy #5: INO-3107 (Therapeutic Vaccine for RRP)

                                          • Targets HPV 6 and 11, which cause recurrent respiratory papillomatosis (RRP).
                                          • Patients can require several surgeries per year to remove papillomas.

                                            • [17:34] — Vaccine Mechanism

                                            • DNA plasmid (vs viral vector in PAP).
                                            • Lower insertional oncogenesis risk.
                                            • Potentially improved durability of response.

                                              • Regulatory Status

                                              • Manufacturer aims to finish BLA by end of 2025.
                                              • Expected approval (with priority review): mid–late 2026.

                                                • [19:17] — Therapy #6: Clemidsogene lanparvovec (Intracerebral Gene Therapy for Hunter Syndrome)

                                                • One-time treatment for MPS II (Hunter syndrome).
                                                • Designed to deliver therapy directly into the brain to address neurological progression.

                                                  • [21:21] — Competing Agent Emerging

                                                  • New enzyme therapy tivedenofusp alfa crosses the blood-brain barrier — unlike current ERT (idursulfase).
                                                  • Expected approval: April 5, 2026.
                                                  • Raises payer & clinical sequencing questions:
                                                    • One-time gene therapy vs repeat annual enzyme therapy
                                                    • Durability vs cost (ERT ~$500K/year)

                                                      • Regulatory Status

                                                      • Clemidsogene lanparvovec anticipated approval: February 8, 2026.

                                                        • [22:45] — Cost, Value, and Durability Considerations

                                                        • Gene therapies may reach multi-million-dollar cost brackets.
                                                        • Durability becomes critical for payer adoption.
                                                        • Hospitals face long onboarding timelines for advanced therapies and must choose which to implement.

                                                          • [22:56] — Where to Learn More

                                                          • Spend Management Outlook (formerly Pharmacy Market Outlook)
                                                          • Oncology and pediatric Vizient therapeutic web pages
                                                          • FDA cell/gene therapy webpage
                                                          • CE session on therapeutic vaccines (Jan 29)

                                                            • [23:46] — Closing

                                                            • Carolyn thanks Amanda.
                                                            • Reminder to subscribe, send comments, and explore further resources from Vizient.

                                                              •  

                                                              Links | Resources: 

                                                              • Spend Management Outlook: Click here
                                                              • Therapeutic class insights: Click here
                                                              • FDA webpage: Click here

                                                                •  

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