![CRISPR CRISPR](https://static.packgene.com/wp-content/uploads/2023/10/CRISPR.jpg)
Intellia Therapeutics has Regeneron in its corner. (Marcos Silva/iStock/Getty Images Plus)
Massachusetts-based Intellia worked with sites in Europe and New Zealand to study the drug candidate, NTLA-2001, in a phase 1 trial. Having shown the candidate may safely reduce levels of misfolded TTR, a protein that drives the rare, life-threatening disease ATTR amyloidosis, the biotech has secured the FDA’s blessing to include U.S. sites in an upcoming phase 3 trial.
Intellia is aiming to start the global phase 3 trial by the end of the year, keeping it on track to achieve the timeline it set out in August. In a statement, Intellia CEO John Leonard, M.D., said the planned study will mark “the first in vivo CRISPR-based candidate to begin late-stage clinical development.” Editas Medicine paused its lead in vivo candidate, EDIT-101, after reporting phase 1/2 data 11 months ago.
The statement about the FDA clearance lacks details of the design of the NTLA-2001 phase 3 trial. David Lebwohl, M.D., chief medical officer at Intellia, gave investors a rough idea of what to expect on a conference call in August, explaining that it will be a “pretty similar” size to Alnylam’s HELIOS-B study.
Alnylam has enrolled 655 ATTR amyloidosis patients in HELIOS-B, a placebo-controlled phase 3 trial that is designed to validate vutrisiran in the indication. The RNAi therapeutic is one of several molecules that are in development or approved for use in ATTR amyloidosis.
BridgeBio Pharma shared phase 3 data on its candidate, acoramidis, earlier this year, while Ionis Pharmaceuticals, Pfizer and other companies are also active in the space.
Intellia has Regeneron in its corner. Under the terms of the partnership, Intellia is taking the lead on the development and commercialization of NTLA-2001, and Regeneron is covering around 25% of the costs in return for the same share of the profits.
Source: https://www.fiercebiotech.com/biotech/intellia-gets-fda-ok-trial-vivo-crispr-therapy-us-teeing-23-start-phase-3-trial
![GMP mRNA](https://static.packgene.com/wp-content/uploads/2023/07/13.jpg)
Check out our AAV CDMO service to expedite your gene therapy research
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
Exploring Tau Protein’s Role in Glaucoma: New Insights and Therapeutic Potential
Glaucoma, a chronic neurodegenerative disorder, leads to irreversible vision loss by damaging retinal ganglion cells (RGCs) and the optic nerve, often associated with increased intraocular pressure (IOP). Despite the benefits of IOP-lowering treatments, the underlying...
FDA-mandated CAR-T monitoring period could be halved, say researchers
In patients with diffuse large B-cell non-Hodgkin lymphoma (DLBCL), the two hallmark post-chimeric antigen receptor (CAR)-T therapy toxicities are extremely rare after two weeks, supporting a shorter, more flexible toxicity monitoring period, according to a study...
Ancestral CRISPR-Cas13 Ribonucleases Discovered: Implications for Genome Editing
In a pioneering study published in *Science*, a team of researchers led by Peter H. Yoon and Jennifer A. Doudna from the University of California, Berkeley, has made a remarkable discovery in the realm of CRISPR technology. The team has identified an ancestral clade...
KBI Biopharma Expands Manufacturing Contract with Global Pharmaceutical Company
KBI Biopharma Inc., a JSR Life Sciences company and global cGMP contract development and manufacturing organization (CDMO), has extended and expanded its manufacturing contract with a leading global pharmaceutical company. Originally initiated in 2020, the renewed...
Related Services
![](https://static.packgene.com/wp-content/uploads/2023/01/9-1.png)
Plasmids GMP Services
![](https://static.packgene.com/wp-content/uploads/2023/01/10-2.png)
AAV GMP Services
![aav icon](https://static.packgene.com/wp-content/uploads/2023/01/9-1.png)