Genevant Sciences Ltd., a new Cambridge, Mass.-based venture with $37.5 million in committed seed funding from Roivant Sciences Ltd., is aiming to get five to 10 RNA-based therapies into the clinic by 2020 using technology from Vancouver, British Columbia-based Arbutus Biopharma Corp. The jointly owned company will develop RNAi, mRNA and gene editing candidates in-house, a departure from Roivant's typical in-licensing strategy. Genevant will focus on a wide range of genetic diseases, both orphan and non-orphan, Roivant spokesman Paul Davis told BioWorld.

Magenta Therapeutics Inc. has closed a $52 million series C financing that it said will be used to advance its portfolio of bone marrow transplantation therapies. The Cambridge, Mass.-based company is testing its most advanced candidate, MGTA-456, in a phase II trial for patients with inherited metabolic diseases.

Less than a year after landing the first gene therapy approval in the U.S. and just months after licensing certain rights to another, Novartis AG is making yet further bets on the field with an $8.7 billion offer to buy Chicago-based Avexis Inc., a company developing a gene therapy for spinal muscular atrophy (SMA).

Sigilon Therapeutics Inc., a Cambridge, Mass.-based startup working to encapsulate therapeutic cells for chronic disease inside microspheres invisible to the immune system's normal fibrotic response to foreign implants, has landed a $473 million deal with Eli Lilly & Co. to develop a potential treatment of type 1 diabetes (T1D).

Sigilon Therapeutics Inc., a Cambridge, Mass.-based start-up working to encapsulate therapeutic cells for chronic disease inside microspheres invisible to the immune system's normal fibrotic response to foreign implants, has landed a $473 million deal with Eli Lilly & Co. to develop a potential treatment of type 1 diabetes (T1D). The strategic collaboration, which includes $63 million up front plus an equity investment of undisclosed value, marks one of Lilly's biggest insulin-dependent diabetes deals to date, as measured by disclosed initial value, after ill-fated tie-ups with Macrogenics Inc. and Adocia SAS.

Aided by regulators' buy-in on an interim endpoint that may hold the key to accelerated approvals in the U.S. and Europe, Retrophin Inc. is kicking off a pivotal phase III test of sparsentan, a potential treatment for the rare kidney disorder focal segmental glomerulosclerosis (FSGS). With the first patient enrolled, the company will pay licensor Ligand Pharmaceuticals Inc. a $4.6 million milestone.

Shares of Sellas Life Sciences Group Inc. (Nasdaq:SLS) jumped 151 percent to close at $8.65 on Monday after the company said an interim analysis of an exploratory phase IIb study seeking the right patient population for its cancer immunotherapy, Neuvax (nelipepimut-S), showed it working synergistically with Herceptin (trastuzumab, Roche Holding AG), making a "clinically meaningful difference" in median disease-free survival for women with triple-negative breast cancer (TNBC).

A top-line win for Pfizer Inc.'s tafamidis, a small-molecule drug in phase III testing to treat a rare and fatal condition associated with progressive heart failure, has shaken up competitive outlooks for Alnylam Pharmaceuticals Inc. and Ionis Pharmaceuticals Inc., both of which are developing alternate therapies in the space. The ATTR-ACT trial, in transthyretin cardiomyopathy, found tafamidis provided a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations vs. placebo at 30 months. It was the first pivotal trial to test a pharmacological treatment for the condition.

Medicinova Inc.'s ibudilast, an experimental anti-inflammatory and neuroprotective agent that has undergone tests in a wide array of neurological conditions, has failed to help addicts kick methamphetamine (MA) dependence in a phase II study, despite early evidence suggesting that it might.