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4BIO Capital publishes review of AAV gene therapies in Cell & Gene Therapy Insights

LONDON & BOSTON – 4BIO Capital (“4BIO” or “the Group”), an international venture capital firm focused solely on the advanced therapies sector, announces the publication of a review paper entitled “Addressing the current limitations of AAV gene therapies” in BioInsights’ Cell & Gene Therapy Insights. The review, written by 4BIO’s Dmitry Kuzmin, Managing Partner and Natalie Johnston, Investment Analyst, focuses on the current limitations of AAV gene therapies and what the sector needs to do to unlock the potential of the approach.

Dmitry Kuzmin, Managing Partner at 4BIO Capital, said: “In vivo gene therapy of human disease using adeno-associated virus (AAV) as a vector has become an established therapeutic modality in the past six years. With three approved drugs and a substantial number of Phase 3 clinical trials, the sector is progressing rapidly to take its place in mainstream medicine. However, critical technological limitations have kept the approach confined to a relatively narrow spectrum of indications thus far.

“In this review we explore the direction of travel towards the follow-on gene therapies that have the potential to expand the reach of the platform towards broader and more complex indications.”

Natalie Johnston, Investment Analyst at 4BIO Capital added: “Whilst recognising that the ability to re-administer and manufacture at scale are two major limitations of AAV therapy, having already been discussed in detail in other publications, this is not the focus of the paper. Instead the review seeks to highlight the ongoing efforts to expand the repertoire of tissues addressable with AAV gene therapy; circumvent the limitations of AAV carrying capacity; and introduce logic and control mechanisms into in vivo gene therapies.”

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