Gene-edited T cells for diabetes treatment

The Scientists at the Seattle Children’s Research Institute and the Benaroya Research Institute have got a promising finding that will turn off the destructive autoimmune response in Type 1 diabetes. This comprises editing the genes of patients’ T cells.

The team presented that by gene-editing CD4+ T cells to express a protein called FOXP3, they could transform the cells into cells having the immunosuppressive properties, as published in the journal Science Translational Medicine. The researchers further engineered the cells to be antigen-specific from T cell receptor (TCR) technology, which has been discovered as an anti-cancer treatment.

They stated at a recent meeting of the American Society of Gene and Cell Therapy that the engineered cells facilitated prevent Type 1 diabetes in mice. In Type 1 diabetes, the body’s T cells erroneously attack insulin-producing beta cells in pancreatic islets.

Pliant Therapeutics raises USD 144 Million IPO 

Pliant Therapeutics raised USD 144 million that will propel its lead asset through mid-stage trials in fibrotic liver and lung diseases and bolster the preclinical development of its earlier-stage programs.

The company initially filed to raise USD 86 million in its IPO last month and doubled its share recently. It also took USD 10 million in a private placement from its partner, Novartis paid USD 80 million in October for a license to Pliant’s preclinical nonalcoholic steatohepatitis (NASH) treatment.

Pliant designated about USD 90 million for its most advanced program, PLN-74809, in development for idiopathic pulmonary fibrosis (IPF) and primary sclerosing cholangitis (PSC). The drug prevents a pair of integrins, alpha V beta 1 and alpha V beta 6 that trigger TGF-beta, a protein, which promotes fibrosis, or scarring, in the lung and bile ducts.

Accent, AstraZeneca aim RNA modifiers in cancer deal

Accent Therapeutics has been discovering new targets for cancer drugs in the space of RNA-modifying proteins. The biotech raised USD 63 million round and having a partnership with AstraZeneca.

Accent is acquiring USD 55 million upfront in discovery, development and commercialization pact. However, it stands to pick up as much as USD 1.1 billion in option fees and milestone payments. Under the agreement, the biotech will take care of R&D work for a nominated preclinical program through the end of phase 1, after that, AstraZeneca will take over clinical development and commercialization activities. The Big Pharma can license two more programs, for which Accent will have to do some preclinical work.

Accent is developing small molecules against RNA-modifying proteins (RMPs) that change the DNA and its functions. The company contemplates that there are many hundreds of RMPs and is working for comprehending the function they play in cancer and which patients would be most likely to respond to its treatments.

Novo, Sanofi lead USD 55 Million round for NodThera

NodThera is setting its aim at the clinic. It is receiving USD 55 million for the round to get its lead asset through phase 1 and a second program into the clinic.

The Cambridge, U.K.-based biotech set up in 2016 to pursue drugs that prevent the NLRP3 inflammasome, a protein complex that has a role in the body’s defence against pathogens. NLRP3 stimulates an inflammatory response that releases cytokines to fight infection. However, it can lead to diseases like nonalcoholic steatohepatitis and diabetes if the process goes wrong.

NodThera is working on drugs, which address NLRP3 that is irregularly activated, raising USD 40 million in 2018 to follow its mission.

Novo Holdings, Cowen Healthcare and Sanofi Ventures have collaborated backers of NodThera in the USD 55 million series B since then, and the company has moved its lead program, NT-0167, into phase 1. It has also discovered compounds that can cross the blood-brain barrier, which could come in handy against diseases like Alzheimer’s and amyotrophic lateral sclerosis, where brain inflammation is involved.