Genetically-Modified AAV and HSCT are the Gene Therapies Leading the Way in Market Expansion of Advance Therapy Biologics
Drug approvals in the Advance Therapy Biologics using gene therapy approaches are accelerating - 22 in total over the last 10 years, with nearly 80% of these having occurred in the last 4 years. This category of BLA approvals focus on genetically-modified viral vectors and cellular products. Since 2022, HSCT Cellular and AAV Viral Vector have dominated the approvals for gene therapy biologics.
* first 6 months of 2024
Viral and Cellular Therapies
HSCT Cellular gene therapies most often involve the use of Hematopoietic Stem Cells (HSCs) that have been autologous-derived and genetically-engineered to express a missing protein. Gene therapies with Hematopoietic Stem Cell Transplants (often acronymed as “HSCT” , or in FDA parlance “autotemcel”) are rapidly expanding. Starting in 2022, 5 HSCT therapies hit the market. Yet, in this same time frame, gene therapies for AAV Viral Vector with Adeno-associated Virus (AAV) have been introduced (FDA parlance “-parvovec” and other “-vec”s, which use a non-replicating, non-integrating virus to infect target cells with a missing gene and of which 7 therapies have been released to market. The remaining 9 therapies hitting market since 2015 are with alternative genetically-modified vectors or cell types (HSV and T-cell).
HSCT Cellular Therapies (4 disease states targeted)
AAV Viral Vector Therapies (6 disease states targeted)
Remaining Gene-modified Viral/Cell Therapies
Bottomline:
Advanced Therapy Biologics using genetically-engineered AAVs and HSCTs are now dominating the BLA approval pipeline.
When do a quick scan of clinicaltrials.gov for interventional studies that are active, recruiting, or soon to be recruiting, we get:
542 studies for Gene Therapy
Then if we restrict that to “Adeno-Associated Virus”
102 studies in AAV
Or restrict to “Hematopoietic Stem Cell”
61 studies in HSC
As a result, a variety of HSC/AAV clinical trials for genetic disorders are hitting the clinic and may soon be approved for use in patients.
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