Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels.
Researchers in the U.K. have succeeded in reverse engineering the defective cryptic splicing that drives amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) to enable precisely targeted delivery of transgenes and therapeutic protein expression in diseased neurons. The technique is compatible with conventional adeno-associated viral vectors that are approved for gene therapy, and can readily be adapted for different transgenes. ALS, FTD and other neurogenerative diseases are underpinned by loss of function of the RNA-binding protein TDP-43 (transactive response DNA-binding protein 43), that normally functions as a key regulator of splicing, protecting the transcriptome from toxic cryptic exons.
Yoltech Therapeutics Co. Ltd. licensed its PCSK9-targeting gene editing therapeutic, YOLT-101, to Shenzhen Salubris Pharmaceuticals Co. Ltd. for mainland China rights in a deal worth ¥1.035 billion (US$145 million).
Yoltech Therapeutics Co. Ltd. licensed its PCSK9-targeting gene editing therapeutic, YOLT-101, to Shenzhen Salubris Pharmaceuticals Co. Ltd. for mainland China rights in a deal worth ¥1.035 billion (US$145 million).
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.
Ending a late 2021 deal that was potentially worth billions, Moderna Inc. and Metagenomi Inc. are going their separate ways. The two had been collaborating on gene-editing R&D to develop therapies for treating serious genetic diseases. Moderna said it agreed with Metagenomi to end the deal as “Moderna continues to strategically prioritize its research and development investments.”
Seven years after embarking on in vivo therapeutic development using CRISPR/Cas gene-editing technology with Intellia Therapeutics Inc., Regeneron Pharmaceuticals Inc. is bringing another company into the collaborative fold. Regeneron will pay Mammoth Biosciences Inc. $100 million, including $95 million as an equity investment, and an up-front payment. Mammoth also could bring in up to $370 million for each target in milestones along with royalties on net sales from products created through the collaboration.
