Your Missing Links Are Here (9 April 2021)
By: Karen O'Hanlon Cohrt - Apr. 9, 2021
- The US National Institutes of Health (NIH) somatic cell gene editing consortium published a progress update on its nationwide effort to develop better gene-editing tools for therapeutic use in Nature journal yesterday. CRISPR Medicine News interviewed one of the lead authors, Erik Sontheimer, to hear more about the consortium's plans.
- The CRISPR patent dispute continues. Read the latest updates here.
- Scribe Therapeutics recently raised $100M in Series B funding to advance its “CRISPR by Design” platform. Scribe Therapeutics was co-founded by Nobel Laureate Jennifer Doudna, and the money will be used to advance the company’s suite of custom gene-editing and delivery technologies, and its pipeline of breakthrough genetic medicines for neurodegeneration and other diseases with high unmet need. Learn more about Scribe in our interview with the CEO last year.
- Cardea Bio introduces SNP-Chip, a CRISPR-based transistor that can detect single point mutations in two diseases so far: amyotrophic lateral sclerosis (ALS) and sickle cell disease (SCD). SNP-Chip, a CRISPR-based gFET system, is an improvement on its predecessor, CRISPR-Chip which we discussed with Cardea's CEO Kiana Anan last year. The research findings behind SNP-Chip were published in Nature Biomedical Engineering this week.
- Metagenomi, a US-based biotech exploring natural microbial communities to discover and develop next-generation therapeutics closed a Series A funding round yesterday with $75 million to support company expansion and development of its therapeutic gene-editing pipeline.
- Inscripta announces the first commercial shipment of its Onyx™ digital genome engineering platform and closes a new financing round with $150 million. Onyx is a high throughput benchtop platform for CRISPR-based genome editing and analysis. The synthetic biology foundry GeneMill at the Universtiy of Liiverpool is the first European player to receive the Onyx platform.
- Researchers in India have developed a tunable doxycycline (dox)-inducible Cas9 expression system for genome editing in iPSC lines using virally delivered Cas9 and gRNAs. The findings were published in Methods in Molecular Biology today.
Clinical trials and regulations
- The American Food and Drug Adminstration (FDA) has approved the first trial for a CRISPR that aims to directly repair the underlying genetic defect in sickle cell disease (SCD). See our earlier piece for a roundup of gene-editing approaches to SCD.
Conferences and webinars
- On 21st April 2021, CRISPR Medicine News will host its very first webinar and attendance is open to all. The webinar’s two speakers will explore the topic ‘Certainty after CRISPR’, looking at the expected and unexpected events that may occur during CRISPR experiments. Find out more and register to attend here.
- Registration is still open for the annual CRISPR conference to be held virtually in June 2021. Abstract submission closes at the end of next week. Find programme and registration details here.
- Genetic conditions rewire our bodies - but science is learning to unpick them. In this episode of RAZOR Science Show, Frankie McCamley speaks to Dr Leslie Gordon, founder of the Progeria Research Foundation that led the discovery of the exact mutation behind progeria, opening the door for possible gene therapies. Watch the episode here. David Liu's group at Broad institute of MIT and Harvard has made major advances towards curing progeria with base editing technology. Check out our recent interview with David Liu to learn more about this work.
- Non-profit organisation Parent Project has granted $31,500 to the University of Missouri School of Medicine to continue evaluating CRISPR therapy in a pre-clinical model of Duchenne. The research, led by Professor Dongsheng Duan will study immune responses from systemic delivery of AAV-mediated CRISPR-Cas9 in a large pre-clinical model. Learn more about gene-editing approaches to Duchenne in our recent overview.
- Charity organisation Fight for Sight is funding cutting-edge gene-editing research at University College London, which will undertake the first study to investigate direct CRISPR-based repair of disease-causing mutations in photoreceptor cells in human models of inherited retinal diseases such as Stargardt disease, retinitis pigmentosa and Leber’s congenital amaurosis.
Podcasts and interviews
- David Liu, Professor at Harvard, the Broad and Howard Hughes Medical Institute talks The Sheekey Science Show about th future of CRISPR (base & prime) and epigenome editing. Watch the recording here.
- Walter Isaacson, author of recent book ‘The Code Breaker’ talks to Ezra Klein about the implications of humanity’s awesome, terrifying takeover of evolution in last week’s Ezra Klein Show. Listen in here or read the podcast transcript here.
- CRISPR Therapeutics CEO Samarth Kulkarni joins the Bid podcast to discuss how the company has advanced clinical trials for CRISPR-based therapies for sickle cell disease, beta thalassemia and cancer. Listen in here.
- Review: A short overview of CRISPR-Cas technology and its application in viral disease control. The applications for CRISPR-Cas systems in controlling mammalian viral infections are discussed.
- Review: Molecular Switch Engineering for Precise Genome Editing. Recent efforts to design "switching" systems for precise control of gene-editing tools are discussed.
- Review: Novel genome-editing-based approaches to treat motor neuron diseases: promises and challenges. The current efforts to tackle motor neuron disease with gene-editing are presented and discussed.