Each month, MabDesign’s market analyses give you an overview of market dynamics and projects in development for a given indication or technology, to help you better understand the constantly evolving market for biomedicines and biomanufacturing. This month, an overview of the gene therapy market and projects currently in development.
Gene therapies, which are part of advanced therapy medicinal products, aim to introduce, repair or replace genes in the patient’s cells. They can be used to treat diseases by modifying the genetic material of cells, offering a targeted approach to correcting the genetic abnormalities that cause various conditions. They open the way to innovative treatments for complex pathologies ranging from cancer to neurological disorders.
Market
In vivo gene therapy drugs (excluding genetically modified cell therapies, such as CAR-T) are few in number on the market: there are currently only 12 products on the market. This number has not changed since our last analysis in March 2024.
Adeno-associated vectors (AAVs) are the main delivery method for gene therapies, accounting for 60% of the products available on the market. AAVs are the vectors of choice because of their tissue tropism, low immunogenicity and genotoxicity, and efficient transduction with sustained gene expression. Their bioproduction has therefore become a major challenge, due to the strong growth in the gene therapy market.
The market was estimated to be worth 4.5 billion dollars in 2023, and is forecast to continue growing strongly, with a CAGR of 20% until 2030.
Pipeline
The prediction of strong market growth over the coming years is based on a very rich pipeline, with 2,135 unique products in development at the end of 2024 (up 28 projects since our last analysis in March 2024). Viral vectors, including AAVs, are chosen as the mode of delivery in 75% of these development projects.
The therapeutic areas targeted by the gene therapies in the pipeline are relatively varied, with a predominance of applications targeting the central nervous system, but also numerous projects in ophthalmology, oncology, and for the treatment of genetic diseases and metabolic disorders.
In terms of the progress of these projects, 85% of the 2,283 projects in development are very early, in the non-clinical phases of development. Only 15% of projects are currently in the clinical phase, which nevertheless represents more than 300 projects.
Therapeutics areas
*unique products
Delivery modes
No Data Found
Development phases (number of projects)
Want to know more ?
MabDesign has acquired and cultivated expertise in all market segments associated with biomedicines and biomanufacturing. Our team of consultants is available to support you in all your projects related to the biotherapeutics sector. Please feel free to explore our range of services, including our marketing and strategic analyses, if you’d like to find out more!
Sources : MabDesign & GlobalData
Date – December 2024
