Gene Therapy Landscape

Since 2004, 32 gene therapies, including genetically modified cell therapies, have been registered covering oncology indications, including non-Hodgkin’s lymphoma (NHL), acute lymphocytic leukaemia (ALL), and chronic lymphocytic leukaemia (CLL). Key players have mainly been top pharma, although Bluebird Bio biotech tops the list with 4 assets (Figure 1 and Table 1).

Figure 1: Top players (by number of approved gene therapies)

Source: Pharmaprojects

Table 1: Most prevalent diseases targeted by approved gene therapies

Source: Pharmaprojects

Eight of these treatments had been approved for the first time since the beginning of 2023 alone for a range of oncology and rare indications (Table 2). CRISPR Therapeutics / Vertex’s Casgevy became the first CRISPR-edited therapy approved in November 2023, specifically for sickle cell anaemia and transfusion-dependent beta thalassemia. The approach consists of genetically-modified autologous CD34+ cells containing haematopoietic stem and progenitor cells edited ex vivo by CRISPR gene editing technology. Another treatment for sickle cell anaemia, Lyfgenia, provides a functional beta-globin gene to a patient’s own haematopoetic stem cells, was approved in December 2023.

Table 2: Gene therapies approved since the beginning of 2023

Source: Pharmaprojects

The pipeline stage is also progressing with 2,092 assets in preclinical – pre-registration. Within late-stage (Phase 3 – Pre-registration), 38 candidates have been identified with smaller players Regenxbio and Ultragenyx Pharmaceutical leading the way with 4 assets each, whilst Novartis and Pfizer have a couple a piece. Most prevalent indications include solid cancer, bladder, and brain cancer.

Figure 2: Top players (by number of gene therapies in
Phase 3 – pre-registration)

Source: Pharmaprojects

Overall, a combination of both large pharma and biotech top the list of players with the highest volume of gene therapies, with Roche capping the charts with 28 assets, followed by AstraZeneca and Sarepta with 21 each (Figure 3). With approved treatments under their belt, Bluebird and CARsgen also have significantly sized portfolios. The majority of diseases included solid cancer, myeloma, acute lymphocytic leukaemia and non-Hodgkin’s lymphoma (Table 3).

Figure 3: Top players (by number of gene therapies
(preclinical – approved)

Source: Pharmaprojects

Table 3: Most prevalent diseases targeted by gene therapies
(preclinical – approved)

Source: Pharmaprojects