The Amazon of Gene Therapy

How scientists used genetic engineering and ML to bring a virus back from the dead as a better deliver method for gene therapies

Molecular structure of the Anc80 virus. [Figure 2C, Zinn et al. 2015]

Delivering Gene Therapy: A Very Brief History (1985–2015)

Delivering genetic packages is hard, but nature has given us a bespoke cellular infiltration system: the adenovirus.

The Lazarus Effect: Inventing Anc80 (2015)

In 2015, the scientist Eric Zinn, along with colleagues from Harvard Medical School and the Massachusetts Eye & Ear Infirmary, published a breakthrough.

Green is Good: green shows the amount of a gene that reached its target when delivered to liver (top row), muscle (middle row) and retina (bottom row) using Anc80 (right column) versus AAV2 (left column) and AAV8 (middle column). Anc80 is better because more of the gene reached its target. [Figure 4A, Zinn et al. 2015]

Preclinical R&D: Delivering Results (2015–2019)

Zinn’s colleagues began testing Anc80 against genetic diseases, starting with challenging disorders of the inner ear.

Towards the Clinic: What’s Next? (2020+)

Biotech companies have begun to develop Anc80 for human patients.

Conclusion: More to Come

From its unique genesis to strong preclinical data, Anc80 is a useful case study in the discovery and development of novel science.

Principal at RA Capital, on the boards of 7 biotech companies. Founder/CEO of Nivien Therapeutics. Australian-American. 50 countries and counting.

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