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Gene Therapy: ‘Words don’t do the results justice’

In 2020, Jake Ternent made history by becoming the first person in the United Kingdom to receive a gene therapy for his inherited retinal disorder.

Having spoken at last year’s Gene Therapy for Ophthalmic Disorders Summit, we sat down with Jake a few weeks ago to discuss how, amid the chaos of a COVID-19 pandemic and ensuing lockdown, 2020 was a turning point in his life.

Grab your copy of this exclusive interview below.

AAV Durability: The Road to a One-Shot Cure

Inconsistent long-term clinical data has highlighted the need to better understand the durability of AAV gene therapy.

Hanson Wade Intelligence's Market Research division have classified 121 AAV gene therapy trials into 4 distinct groups based on the target tissue type – liver, muscle, CNS and ocular tissues. This allowed us to summarise available data on clinical durability as well as the key findings we observed across tissues, trials, doses and serotypes.

We hope that this report will provide gene therapy developers with valuable insights on the durability of the current generation of AAV gene therapies.