CRISPR: When Gene Editing Became Medicine
CRISPR sat in laboratories for more than a decade - a bacterial immune system, then a programmable tool - before anyone was treated with it. This video follows that gap: from the 2000s discovery that bacteria keep a genetic memory of the viruses that attack them, through the 2012 paper that showed the cutting enzyme Cas9 could be redirected to any DNA sequence, to the first patient treated in 2019 and the first approved CRISPR medicine, Casgevy, in December 2023.
It explains how the edit actually works - guide RNA, Cas9's cut, and the newer, gentler base-editing method that changes one DNA letter without cutting at all - and what had to be solved before any of it could reach a patient safely: off-target cuts, delivery into the right cells, and manufacturing at hospital scale.
Then it follows the list of what gene editing can treat as it starts to grow: a base-edited leukaemia cure in London in 2022, Casgevy's extension to a second blood disorder in 2024, and a bespoke, one-patient therapy built for an infant, KJ Muldoon, in just six months in 2025. Against that, it follows where the approach still breaks - an in-body CRISPR trial placed on FDA clinical hold in late 2025 after a patient died - and the FDA's January 2026 attempt to make its own manufacturing review faster.
It closes by weighing a small, approved, expensive list of cures against the more than ten thousand other genetic diseases gene editing cannot yet touch.
Educational documentary. Not financial or investment advice.
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Sources and credits
Photo credits (Wikimedia Commons)
- Jennifer Doudna, CRISPR-Cas9 co-discoverer: The White House, Public domain - https://commons.wikimedia.org/wiki/File%3AP20230620AS-0276_%2853233832252%29.jpg
- University of California, Berkeley campus (Doudna's university): Simon Cobb, CC0 - https://commons.wikimedia.org/wiki/File%3ACampus_of_the_University_of_California.jpg
- Kendall Square, Cambridge MA (Vertex/CRISPR Therapeutics biotech corridor): Tim Pierce, Public domain - https://commons.wikimedia.org/wiki/File%3AKendall_Square.jpg
- FDA White Oak campus, Silver Spring MD: The U.S. Food and Drug Administration, Public domain - https://commons.wikimedia.org/wiki/File%3AFDA_Bldg_62_-_Exterior_%285161375340%29.jpg
Primary sources
- Mojica, Diez-Villasenor, Garcia-Martinez, 'Intervening Sequences of Regularly Spaced Prokaryotic Repeats Derive from Foreign Genetic Elements', Journal of Molecular Evolution, 2005.
- Barrangou, Horvath et al., 'CRISPR Provides Acquired Resistance Against Viruses in Prokaryotes', Science, 23 March 2007.
- Jinek, Chylinski, Fonfara, Hauer, Doudna, Charpentier, 'A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity', Science, 28 June 2012 (online).
- NobelPrize.org, 'The Nobel Prize in Chemistry 2020 - Popular information'.
- NPR, Rob Stein, 'In A First, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder', 29 July 2019, and 'First Sickle Cell Patient Treated With CRISPR Gene-Editing Still Thriving', 31 December 2021.
- FDA, 'FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease', 8 December 2023.
- Vertex/CRISPR Therapeutics investor release, 16 January 2024 (Casgevy beta-thalassaemia approval); Healio and Pharmacy Times coverage of the FDA's paediatric label expansion, July 2026.
- Vertex Pharmaceuticals Q4 2025 earnings results, reported 12 February 2026 (BioPharma Dive coverage) - Casgevy patient-initiation figures.
- Great Ormond Street Hospital/UCL press materials on Alyssa Tapley and BE-CAR7, 2023; ASH 2025 trial-update coverage, December 2025.
- Musunuru et al., 'Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease', New England Journal of Medicine, 15 May 2025; NBC News and CHOP/Penn Medicine press releases, 15 May 2025.
- Intellia Therapeutics, SEC Form 8-K filings, October 2025-January 2026; CGTLive, 9 November 2025; Intellia press release, 27 January 2026 (MAGNITUDE-2 hold lifted).
- FDA, 'Flexible Requirements: Cell and Gene Therapies Advance Innovation', fda.gov, 11 January 2026.
Not regulated financial advice.