Return of Pipeline 2026 – Cell and Gene Therapies cover art

Return of Pipeline 2026 – Cell and Gene Therapies

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 yearMajor reduction in hospitalizationsDiscontinuation 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 effectGraft-versus-infection protectionReduction 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 therapyDurability vs cost (ERT ~$500K/year) Regulatory Status Clemidsogene lanparvovec anticipated approval: February 8, 2026. 22:45 ...
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