19 May 2020

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.

“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.”

Dmitry Kuzmin

Managing Partner at 4BIO Capital

“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.”

Natalie Johnston

Investment Analyst at 4BIO Capital




4BIO Capital

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About 4BIO Capital

4BIO Capital is an international venture capital firm focused solely on the advanced therapies sector.


4BIO’s objective is to invest in, support, and grow early stage companies developing treatments in areas of high unmet medical need, with the ultimate goal of ensuring access to these potentially curative therapies for all patients. Specifically, it looks for viable, high-quality opportunities in cell and gene therapy, RNA-based therapy, targeted therapies, and the microbiome.


The 4BIO team comprises leading advanced therapy scientists and experienced life science investors who have collectively published over 250 scientific articles in prestigious academic journals including Nature, The Lancet, Cell, and the New England Journal of Medicine. 4BIO has both an unrivalled network within the advanced therapy sector and a unique understanding of the criteria that define a successful investment opportunity in this space.


For more information, please visit www.4biocapital.com




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