Gene Therapies — Pipeline, Approvals, Sponsors

In vivo gene-replacement or gene-editing therapeutics. Primarily AAV (adeno-associated virus) and lentiviral vectors. Spark / Zolgensma / Luxturna kicked off the modern wave; pipeline expanding to inherited retinal diseases, hemophilia, sickle cell, DMD.

Marketed leaders

• 2023-01-01 — Casgevy · Autologous genome-edited hematopoietic stem cell therapy
• 2022-01-01 — Roctavian · BioMarin International Limited · Rare Disease
• 2022-01-01 — Upstaza · PTC Therapeutics International Limited · Gastroenterology
• 2020-01-01 — BREXUCABTAGENE AUTOLEUCEL
• 2019-01-01 — BETIBEGLOGENE AUTOTEMCEL
• 2018-01-01 — VORETIGENE NEPARVOVEC
• 2018-01-01 — AXICABTAGENE CILOLEUCEL · CD19-directed Chimeric Antigen Receptor [EPC]
• 2015-01-01 — Imlygic · Amgen · Oncology
• 2012-01-01 — Glybera · uniQure biopharma B.V. · Metabolic
• — Breyanzi · Oncology

Recent approvals (last 30 days)

No approvals in the last 30 days.

Upcoming PDUFA (next 180 days)

No upcoming PDUFA in next 180 days.

Top sponsors in this modality

1. · 8 gene therapies
2. Pfizer Inc. · 3 gene therapies
3. Amgen · 1 gene therapies
4. BioMarin International Limited · 1 gene therapies
5. PTC Therapeutics International Limited · 1 gene therapies
6. uniQure biopharma B.V. · 1 gene therapies

Drug classes within Gene Therapies

• Autologous genome-edited hematopoietic stem cell therapy · 1
• CD19-directed Chimeric Antigen Receptor [EPC] · 1
• Gene therapy; In vivo gene delivery · 1

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Other modalities

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing