Vertex, Mammoth Launch Up-to-$695M CRISPR Gene-Editing Collaboration – Genetic Engineering & Biotechnology News
Posted: October 27, 2021 at 1:43 am
Vertex Pharmaceuticals will develop in vivo gene-editing therapies for two genetic diseases using Mammoth Biosciences CRISPR systems through a collaboration that could generate more than $695 million for Mammoth, the companies said today.
The companies is not disclosing which diseases they will target.
We are focused on developing in vivo gene-editing therapies in two indications for specific serious and/or life-threatening diseases with the Vertex team,Peter Nell, Mammoths Chief Business Officer and Head of Therapeutics Strategy, told GEN.
Mammoth and Vertex did say, however, that they will apply Mammoths CRISPR platform consisting of a proprietary toolbox of novel Cas enzymeswhat the company calls the largest toolbox of CRISPR proteins on earth.
These include Cas12, which targets double-stranded DNA;Cas13, which targets and recognizes single-stranded RNA; Cas14, which targets single-stranded DNA; and Cas, which is encoded exclusively in the genomes of huge bacteriophages.
Mammoth has exclusively licensed foundational IP around novel CRISPR Cas12, Cas13, Cas14, and Cas systems from the University of California, Berkeley, where, they were discovered in the lab of Nobel laureate and Berkeley-based researcher Jennifer Doudna, PhD.
Doudna is a co-founder of Mammoth Biosciencesalong with CEO Trevor Martin, PhD; Janice Chen, PhD, the companys CTO, who discussed her companys technology last year onGENs monthly GEN Live program; Lucas Harrington, Mammoths CSO; and Ashley Tehranchi, PhD, who served as CTO until May 2019.
Cas14 and Cas are the smallest known CRISPR systems. Their sizes530 amino acids for Cas14a and 757 amino acids for Cas-2are less than half those of commonly used variants of Cas9 [1368 amino acids for SpCas9] and Cas12 [1,300 amino acids for FnCas12], offering numerous potential advantages for the therapies Mammoth plans to develop, Martin told GEN Edge last month, after the company announced the completion of $195 million in new financing completed over the past year.
The additional financingconsisting of $150 million in Series D financing and a $45-million Series C round whose investors included Amazonbrought Mammoths total capital raised from investors to more than $255 million., propelling the company to a unicorn valuation of more than $1 billion.
In addition, Mammoth said, it is building out its IP portfolio by discovering novel CRISPR systems within and beyond the foundational work. The company has yet to disclose those systems or other Cas enzymes under development.
The combination of Mammoths unique technology with Vertexs unmatched experience in serious disease research and development will only accelerate programs with the goal of reaching patients with high unmet medical need, Nell added. We believe our novel ultra-small CRISPR systems have the potential to be game-changers when it comes to systemic and targeted delivery of in vivo gene-editing therapies.
CRISPR-edited therapies have been an area of focus for Vertex. Late last year, the company and CRISPR Therapeutics reported positive data from a pair of Phase I/II trials for their CRISPR-Cas9 gene-edited therapy CTX001 showing consistent and sustained positive response in 10 patients treated for a pair of blood disorders, sickle cell disease (SCD) and beta thalassemia.
The companies in April amended their collaboration agreement to give Vertex leadership in global development, manufacturing, and commercialization of CTX001 with support from CRISPR Therapeutics, in return for CRISPR receiving a $900 million upfront payment and a potential additional $200 million milestone payment upon CTX001 regulatory approval. Two months later, during the Joint European Hematology Association-American Society of Hematology (EHA-ASH) Symposium, researchers presented additional clinical data showing CTX001 to have delivered a consistent and sustained response to treatment in 22 patients in two ongoing Phase I/II trials.
We see tremendous potential for CTX001, Stuart A. Arbuckle, Vertexs executive vice president and chief commercial and operations officer, told analysts July 29 on the companys quarterly earnings call following release of second-quarter results. He cited an estimate of more than 150,000 patients in the United States and Europe, who have beta thalassemia, or sickle cell disease, approximately 32,000 of whom have severe disease; plus another 25,000 severe sickle cell disease patients, the vast majority of which were in the United States.
We believe that a gene-editing approach which holds the potential for a one-time curative treatment will be highly valued by patients, physicians, and payers, Arbuckle said. Consistent with our own internal market research, published physician surveys in the United States consistently indicate that they expect a quarter to a third of their sickle cell disease patients would be good candidates for a one-time curative approach using the current conditioning regimen, which is in line with the estimates of the numbers of severe patients.
With gentler conditioning regimens in the future, Arbuckle added, we expect CTX001 to be an attractive option for a much larger proportion of the 150,000 beta thalassemia and sickle cell disease patients.
To launch its collaboration with Mammoth, Vertex has agreed to pay the Brisbane, CA,-based company $41 million upfront, including an investment in the form of a convertible note, and up to $650 million in potential future payments tied to achieving research, development, and commercial milestones across two potential programs.
Mammoth is also eligible for tiered royalties from Boston-based Vertex on future net sales on any products that may result from the collaboration, the first one announced by Mammoth for the development of gene-edited therapies.
Vertex and Mammoth share the same commitment to developing therapies that have the potential to be transformative for people with serious diseases, stated David Altshuler, MD, PhD, Vertexs CSO. We look forward to expanding our cell and genetic therapies capabilities with the addition of Mammoths ultra-small CRISPR systems for in vivo genome editing, which will provide us with another set of tools to tackle many of the diseases were interested in.
Mammoth is also developing CRISPR-based diagnostics, having applied Cas12 in itsCOVID-19diagnostic effort which culminated in the SARS-CoV-2 RNA DETECTR Assay, a COVID-19 diagnostic for whichUCSF Health Clinical Laboratorieswasgranted an FDA Emergency Use Authorization (EUA)in August 2020.
The 45-minute test is designed to detect nucleic acid from SARS-CoV-2 in upper respiratory specimens. The test extracts, isolates, and purifies SARS-CoV-2 nucleic acid for simultaneous reverse transcription into cDNA, followed by amplification using loop-mediated amplification (RT-LAMP).
The SARS-CoV-2 RNA DETECTR Assay was co-developed by Mammoth through itspartnership with UCSF professor Charles Chiu, MD, PhD, who is also director of the UCSF-Abbott Viral Diagnostics and Discovery Center, and a member of the companys Scientific Advisory Board. Mammothin 2019exclusively licensed two U.S. patents granted to the regents of the University of California that cover CRISPR collateral cleavage diagnostic systems.
In July 2020, Mammoth won funding for its development of a scalable COVID-19 test, when the company wasawarded $23.1 millionof $248.7 million in contracts to the first seven lab-based and point-of-care tests diagnostics developersfunded through the NIHs Rapid Acceleration of Diagnostics (RADx) initiative. The testing system can be adapted to detect for other viruses, though Mammoth has not made public which ones.
Two months earlier in May 2020, Mammoth launched a collaboration with GlaxoSmithKlines GSK Consumer Healthcare to develop a handheld test designed to apply the DETECTR platform at point of need. Mammoth has disclosed few details since the initial announcement, with Martin saying last month: I cant say too much about it, but definitely weve made huge strides.
Original post:
Vertex, Mammoth Launch Up-to-$695M CRISPR Gene-Editing Collaboration - Genetic Engineering & Biotechnology News
- 'CRISPR pill' instructs harmful bacteria to self-destruct - National Hog Farmer [Last Updated On: April 26th, 2017] [Originally Added On: April 26th, 2017]
- Highly sensitive CRISPR diagnostic tool created - BioNews [Last Updated On: April 26th, 2017] [Originally Added On: April 26th, 2017]
- More Tooth, More Tail in CRISPR Operations | GEN - Genetic Engineering & Biotechnology News (press release) [Last Updated On: April 26th, 2017] [Originally Added On: April 26th, 2017]
- Quick, Sensitive Diagnostic Tests with CRISPR - Technology Networks [Last Updated On: April 26th, 2017] [Originally Added On: April 26th, 2017]
- MPEG LA Invites CRISPR-Cas9 Patents to be Pooled in a One-Stop License - Yahoo Finance [Last Updated On: April 26th, 2017] [Originally Added On: April 26th, 2017]
- What Is CRISPR? - livescience.com [Last Updated On: April 26th, 2017] [Originally Added On: April 26th, 2017]
- CRISPR and Stem Cells Identify Novel Chlamydia Drug Targets - Genetic Engineering & Biotechnology News [Last Updated On: April 26th, 2017] [Originally Added On: April 26th, 2017]
- CRISPR webinar: HGF discusses IP landscape - Life Sciences Intellectual Property Review (subscription) [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- CRISPR.com was for sale, and you won't guess who bought it - STAT [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- CRISPR Pill May Be Key in Fight Against Antibiotic Resistance - Singularity Hub [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- Intellia (NTLA), CRISPR Therapeutics (CRSP) Receive U.S. Patent for CRISPR/Cas9 Ribonucleoprotein Complexes - StreetInsider.com [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- transOMIC technologies Launches transEDIT-dual CRISPR ... - PR Newswire (press release) [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- Global CRISPR Market Forecast 2017-2025 - Research and Markets ... - Business Wire (press release) [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- Quick, Sensitive Diagnostic Tests with CRISPR | Technology Networks - Technology Networks [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- CRISPR/Cas9 and Targeted Genome Editing: A New Era in ... [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- CRISPR - Wikipedia [Last Updated On: April 27th, 2017] [Originally Added On: April 27th, 2017]
- CRISPR Used To Modify Multiple Genes In Rice - Asian Scientist Magazine [Last Updated On: April 28th, 2017] [Originally Added On: April 28th, 2017]
- Current CRISPR Patent Dispute, Explained - CALIFORNIA [Last Updated On: April 28th, 2017] [Originally Added On: April 28th, 2017]
- CEOs of top gene-editing firms got huge compensation hikes last year - Boston Business Journal [Last Updated On: April 28th, 2017] [Originally Added On: April 28th, 2017]
- CRISPR-SMART Cells Regenerate Cartilage, Secrete Anti-Arthritis Drug - Genetic Engineering & Biotechnology News [Last Updated On: April 29th, 2017] [Originally Added On: April 29th, 2017]
- Another CRISPR Trial Begins - GenomeWeb [Last Updated On: April 29th, 2017] [Originally Added On: April 29th, 2017]
- China Is Racing Ahead of the US in the Quest to Cure Cancer With CRISPR - Gizmodo [Last Updated On: April 29th, 2017] [Originally Added On: April 29th, 2017]
- CRISPR Gene Editing - CRISPR/Cas9 - Horizon Discovery [Last Updated On: May 1st, 2017] [Originally Added On: May 1st, 2017]
- CRISPR | Broad Institute [Last Updated On: May 1st, 2017] [Originally Added On: May 1st, 2017]
- Questions and Answers about CRISPR | Broad Institute [Last Updated On: May 1st, 2017] [Originally Added On: May 1st, 2017]
- CRISPR Genome Engineering Resources | learn, share, and discuss [Last Updated On: May 1st, 2017] [Originally Added On: May 1st, 2017]
- CRISPR Technology Scientists on Their Gene Editing Tool - TIME [Last Updated On: May 1st, 2017] [Originally Added On: May 1st, 2017]
- Cas9 - Wikipedia [Last Updated On: May 1st, 2017] [Originally Added On: May 1st, 2017]
- Using CRISPR against cancer shows success in mice - Futurity - Futurity: Research News [Last Updated On: May 2nd, 2017] [Originally Added On: May 2nd, 2017]
- Using CRISPR to Find Treatments for Aggressive Pediatric Brain Cancer - Bioscience Technology [Last Updated On: May 2nd, 2017] [Originally Added On: May 2nd, 2017]
- CRISPR Eliminates HIV in Live Animals - Genetic Engineering & Biotechnology News [Last Updated On: May 2nd, 2017] [Originally Added On: May 2nd, 2017]
- The CRISPR patent dispute - Europe and the US - BioNews [Last Updated On: May 2nd, 2017] [Originally Added On: May 2nd, 2017]
- How Scientists Think CRISPR Will Change Medicine - TIME [Last Updated On: May 3rd, 2017] [Originally Added On: May 3rd, 2017]
- What you need to know about the legal battle over CRISPR patents - Genetic Literacy Project [Last Updated On: May 4th, 2017] [Originally Added On: May 4th, 2017]
- Scientists have eliminated HIV in mice using CRISPR - TechCrunch [Last Updated On: May 4th, 2017] [Originally Added On: May 4th, 2017]
- CRISPR Therapeutics Appoints Samarth Kulkarni, Ph.D. as President, Expanding Role Beyond Chief Business Officer ... - GlobeNewswire (press release) [Last Updated On: May 4th, 2017] [Originally Added On: May 4th, 2017]
- ECDC says risk from contaminated CRISPR kits low - CIDRAP [Last Updated On: May 4th, 2017] [Originally Added On: May 4th, 2017]
- CRISPR Could Transform the Way We Diagnose Disease - Gizmodo [Last Updated On: May 4th, 2017] [Originally Added On: May 4th, 2017]
- A cancer gene also grows stem cells, CRISPR in monkey embryo ... - Speaking of Research [Last Updated On: May 5th, 2017] [Originally Added On: May 5th, 2017]
- New CRISPR Technique Can Potentially Stop Cancer In Its Tracks - Wall Street Pit [Last Updated On: May 6th, 2017] [Originally Added On: May 6th, 2017]
- CRISPR gene-editing tool targets cancer's "command center" - Gizmag - New Atlas [Last Updated On: May 7th, 2017] [Originally Added On: May 7th, 2017]
- Update: CRISPR - Radiolab [Last Updated On: May 7th, 2017] [Originally Added On: May 7th, 2017]
- Cambridge gene editing firm CRISPR to use delivery tech honed ... - Boston Business Journal [Last Updated On: May 9th, 2017] [Originally Added On: May 9th, 2017]
- Oxford Genetics licenses CRISPR tech to power synbio push - FierceBiotech [Last Updated On: May 10th, 2017] [Originally Added On: May 10th, 2017]
- What You Need to Know About the New CRISPR Cancer Treatment - BOSS Magazine [Last Updated On: May 11th, 2017] [Originally Added On: May 11th, 2017]
- CRISPR: The Future of Medicine and Human Evolution - in-Training [Last Updated On: May 12th, 2017] [Originally Added On: May 12th, 2017]
- Intellia Therapeutics Announces Progress with CRISPR/Cas9 at the American Society of Gene & Cell Therapy Annual ... - GlobeNewswire (press... [Last Updated On: May 13th, 2017] [Originally Added On: May 13th, 2017]
- Pac-Man like CRISPR enzymes discovered - Lab News [Last Updated On: May 13th, 2017] [Originally Added On: May 13th, 2017]
- Coming age of CRISPR gene editing: What in heck is the 'Pink Chicken Project'? - Genetic Literacy Project [Last Updated On: May 15th, 2017] [Originally Added On: May 15th, 2017]
- Intellia moves closer to clinic with CRISPR tech - FierceBiotech [Last Updated On: May 15th, 2017] [Originally Added On: May 15th, 2017]
- Will CRISPR Technology Create a New "Human" Species? - Big Think [Last Updated On: May 15th, 2017] [Originally Added On: May 15th, 2017]
- Caribou Bioscience's CEO on CRISPR's legal and ethical challenges - TechCrunch [Last Updated On: May 16th, 2017] [Originally Added On: May 16th, 2017]
- Cut Out the Hype: Gene Editing With CRISPR and the Truth about Superhuman 'Designer Babies' - WhatIsEpigenetics.com (blog) [Last Updated On: May 16th, 2017] [Originally Added On: May 16th, 2017]
- CRISPR-Cas.org [Last Updated On: May 16th, 2017] [Originally Added On: May 16th, 2017]
- Synthego's genetic toolkit aims to make CRISPR more accessible - TechCrunch [Last Updated On: May 16th, 2017] [Originally Added On: May 16th, 2017]
- What is CRISPR? A Beginner's Guide | Digital Trends [Last Updated On: May 16th, 2017] [Originally Added On: May 16th, 2017]
- Geneticists Enlist Engineered Virus and CRISPR to Battle Citrus Disease - Scientific American [Last Updated On: May 16th, 2017] [Originally Added On: May 16th, 2017]
- Editas delays IND for Allergan-partnered CRISPR program - FierceBiotech [Last Updated On: May 16th, 2017] [Originally Added On: May 16th, 2017]
- Easy DNA Editing Will Remake the World. Buckle Up - WIRED [Last Updated On: May 16th, 2017] [Originally Added On: May 16th, 2017]
- Can CRISPR feed the world? - Phys.org - Phys.Org [Last Updated On: May 19th, 2017] [Originally Added On: May 19th, 2017]
- Gene-editing tool 'CRISPR' gaining massive attention - KMOV.com [Last Updated On: May 19th, 2017] [Originally Added On: May 19th, 2017]
- Fixing the tomato: CRISPR edits correct plant-breeding snafu - Nature.com [Last Updated On: May 19th, 2017] [Originally Added On: May 19th, 2017]
- Beyond just promise, CRISPR is delivering in the lab today - The Conversation US [Last Updated On: May 20th, 2017] [Originally Added On: May 20th, 2017]
- What is CRISPR-Cas9, and will it change the world? | Alphr - Alphr [Last Updated On: May 20th, 2017] [Originally Added On: May 20th, 2017]
- Fixing the Tomato: CRISPR Edits Correct Plant-Breeding Snafu ... - Scientific American [Last Updated On: May 20th, 2017] [Originally Added On: May 20th, 2017]
- This UK Biotech uses CRISPR-Cas9 To Fight Bacterial Resistance - Labiotech.eu (blog) [Last Updated On: May 21st, 2017] [Originally Added On: May 21st, 2017]
- Can CRISPR feed the world? | Horizon: the EU Research ... - Horizon magazine [Last Updated On: May 21st, 2017] [Originally Added On: May 21st, 2017]
- Will this gene-editing tool cure the diseases of the future? - Sacramento Bee [Last Updated On: May 23rd, 2017] [Originally Added On: May 23rd, 2017]
- How the CRISPR-Cas9 System is Redefining Drug Discovery - Labiotech.eu (blog) [Last Updated On: May 23rd, 2017] [Originally Added On: May 23rd, 2017]
- Scientists are using gene editing to create the perfect tomato for your salad - Quartz [Last Updated On: May 24th, 2017] [Originally Added On: May 24th, 2017]
- Fine-tuning CRISPR to Create Popular Mouse Models - Technology Networks [Last Updated On: May 25th, 2017] [Originally Added On: May 25th, 2017]
- Scientists Are Using CRISPR To "Program" Living Cells - Futurism - Futurism [Last Updated On: May 25th, 2017] [Originally Added On: May 25th, 2017]
- CRISPR gene editing puts the brakes on cancer cells - Cosmos [Last Updated On: May 26th, 2017] [Originally Added On: May 26th, 2017]
- Watch This Scientist Brilliantly Explain CRISPR to Everyone from a Child to a Ph.D. - Patheos (blog) [Last Updated On: May 27th, 2017] [Originally Added On: May 27th, 2017]
- Using CRISPR gene editing to slow cancer growth | FierceBiotech - FierceBiotech [Last Updated On: May 27th, 2017] [Originally Added On: May 27th, 2017]
- How A Gene Editing Tool Went From Labs To A Middle-School Classroom - NPR [Last Updated On: May 27th, 2017] [Originally Added On: May 27th, 2017]
- In Just a Few Short Years, CRISPR Has Sparked a Research Revolution - Futurism [Last Updated On: May 29th, 2017] [Originally Added On: May 29th, 2017]
- CRISPR Is Taking Over Science, Breaks Out Of Labs And Invades Schools - EconoTimes [Last Updated On: May 30th, 2017] [Originally Added On: May 30th, 2017]
- Gene-editing technique scientists hope will cure cancer and all ... - The Independent [Last Updated On: May 30th, 2017] [Originally Added On: May 30th, 2017]
- CRISPR Gene-Editing Can Cause Hundreds of Unexpected ... - ScienceAlert [Last Updated On: May 30th, 2017] [Originally Added On: May 30th, 2017]