Somatic human genome editing has made huge strides in the past five years, but the likely extremely high prices will be unsustainable. A global commitment to affordable, equitable access is urgently needed because the costs and infrastructure needs of this form of treatment are not manageable for either patients or health care systems.
The hexanucleotide repeat expansion (HRE) GGGGCC in the noncoding region of the chromosome 9 open reading frame 72 (C9ORF72) gene is the most common cause of hereditary (40%) and apparently sporadic (5%-6%) amyotrophic lateral sclerosis (ALS).
A lackluster efficacy signal has prompted Editas Medicine Inc. to pause enrollment in a phase I/II trial of its CRISPR/Cas9-based gene editing therapy, EDIT-101, which is in development for patients with a particular form of Leber congenital amaurosis type 10.
Versant Ventures is committing $50 million in series A funding to Cimeio Therapeutics Inc., which aims to bring new possibilities in terms of disease targeting and safety to bear on a wide range of cell therapy applications, including hematopoietic stem cell transplantation (HSCT) and adoptive cell transfer.
Tune Therapeutics Inc. launched with $40 million in initial financing and plans to pursue both rare and complex disease indications with a series of epigenetic editors that employ CRISPR-Cas9 DNA recognition to modulate gene expression in a targeted fashion, without introducing potentially problematic DNA strand breaks or changes to the genetic code.
LONDON – Intellia Therapeutics Inc. is to receive 10% of the equity in Sparingvision SAS as part of a deal giving the French ophthalmology specialist certain exclusive rights to in vivo CRISPR/Cas9 technologies in the treatment of ocular diseases.
CEO John Leonard said Intellia Therapeutics Inc. plans “to share information on a cohort-by-cohort basis, so we get a consistent readout” and, as the year goes on, longer-term follow-up findings will emerge from the phase I trial with the company’s lead in vivo genome editing candidate, NTLA-2001.
In biotech and biopharma’s third-largest ever up-front development and commercialization deal, Crispr Therapeutics AG will receive an initial $900 million in an amended deal with Vertex Pharmaceuticals Inc. to lead the development, manufacturing and commercialization of gene editing therapy CTX-001 for sickle cell disease and transfusion-dependent beta-thalassemia.
Surprising no one, Jennifer Doudna and Emmanuelle Charpentier won the 2020 Nobel Prize in Chemistry “for the development of a method for genome editing,” that is, the CRISPR/Cas9 system.
In case you haven't heard, Tessera Therapeutics Inc. is working on techniques to write genes into the genome of patients. Tessera, which was developed in Flagship Pioneering Inc.'s Flagship Labs and became a stand-alone incorporated company two years ago, recently came out of stealth mode to highlight its Gene Writing platform based on mobile genetic elements, such as transposons and retrotransposons.
