Currently available disease management options for Duchenne muscular dystrophy (DMD) are mostly symptomatic. Several strategies based on exon-skipping or gene transfer have been proposed to restore dystrophin expression, but can only address specific subsets of DMD patients and/or provide limited clinical benefits. Upregulating utrophin (UTRN), a structural and functional paralogue of dystrophin, has been proposed as an alternative therapeutic approach that may be suitable for all DMD patients, regardless of their genetic defect.
Following a clinical hold last October of Intellia Therapeutics Inc.’s Magnitude and Magnitude-2 phase III trials of CRISPR/Cas9 gene editing therapy nexiguran ziclumeran (nex-z) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively, the U.S. FDA lifted the hold on Magnitude-2, pushing the company’s shares up by 22% in early trading Jan. 27.
Toolgen Inc. has entered into a strategic cross-license agreement with Geneditbio Ltd. to jointly advance the development of next-generation in vivo genome-editing therapeutics.
San Francisco Bay Area researchers from UC Berkeley, UC San Francisco and Stanford University have combined their technologies to create Azalea Therapeutics Inc., a company focused on editing cells in vivo.
San Francisco Bay Area researchers from UC Berkeley, UC San Francisco and Stanford University have combined their technologies to create Azalea Therapeutics Inc., a company focused on editing cells in vivo.
Newco Brink Therapeutics SAS is poised to work on the next chapter in genome editing after raising €3.5 million (US$4 million) in seed funding to discover and develop programmable recombinase enzymes.
Newco Akribion Therapeutics GmbH has raised €8 million (US$8.3 million) in a seed round to develop a new and potent class of RNA-targeted CRISPR nucleases, which, rather than cleaving specific nucleic acids, can destroy every type of nucleic acid in a cell.
Newco Akribion Therapeutics GmbH has raised €8 million (US$8.3 million) in a seed round to develop a new and potent class of RNA-targeted CRISPR nucleases, which, rather than cleaving specific nucleic acids, can destroy every type of nucleic acid in a cell.
Forkhead box protein L2 (FOXL2) is a protein involved in ovary development and maintenance, among other functions. Mutations in its gene are involved in ovarian failure and granulosa cell tumors, being the latter a rare cancer type caused in most cases by the C134W mutation (c.402C>G) in the FOXL2 gene. By using CRISPR/Cas9 technology, researchers from Spain have aimed to remove this mutation in the FOLX2 gene from granulosa tumor cells.
Researchers from Excision Biotherapeutics Inc. and affiliated organizations have reported the development of a novel CRISPR/Cas9-mediated genome editing therapy – EBT-104 – for the treatment of latent herpes simplex virus 1 (HSV-1) keratitis.
