Rather than reinventing the wheel for every gene therapy that uses genome editing, the U.S. FDA is advising sponsors on leveraging existing knowledge, be it publicly available or platform-based, to more efficiently advance their products across multiple stages of development.
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has opened a consultation on changes it is proposing to the legal definition of gene therapies, to reflect the advances in technology and manufacturing over the two decades since the current legislation was drawn up. The aim is to correct the mismatch between regulatory terminology and modern science, which MHRA says “can lead to uncertainty” over how a product is classified and to “inconsistent oversight and potential barriers for developers."
Glucocorticoid replacement therapy is the current standard of care for congenital adrenal hyperplasia (CAH). However, new therapeutic strategies that can better recapitulate physiological requirements and reduce morbidity and mortality among CAH patients are urgently needed. Despite the promise of gene therapy for correcting monogenic disorders, the strategies investigated to date have not yielded satisfactory results.
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.
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.
Anges Inc. has entered into a sponsored research agreement with Stanford University School of Medicine for the development of novel cancer therapies using genome editing technology. The parties aim to combine nucleic acid drug delivery technology developed at Stanford with the genome editing technology of Emendobio Inc., a subsidiary of Anges.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria. The advance opens the door to the development of programmable methods for rearranging DNA, using recombinase enzymes guided by RNA. The two different approaches to using insertion sequences (IS) – some of the simplest and most compact mobile genetic elements – are described in two papers published in Nature and Nature Communications.
Genome editing specialist Tome Biosciences Inc. now has all the bases covered, after arriving on the scene in December with $213 million funding and three weeks later announcing the acquisition of fellow precision editing company, Replace Therapeutics Inc. for up to $185 million.
