Due to the need for chemotherapy and the risks caused by allogeneic stem cell transplants in Fanconi anaemia patients, Rocket Pharma has developed gene therapy candidate RP-L102, which doesn’t need conditioning and corrects the gene mutation underlying Fanconi. Rocket CEO Guarav Shah explains RP-L102’s mechanism of action, as well as how the company is working to improve its cell culturing methods and overcome continuing risks of head and neck cancer.

Drug resistance presents a significant challenge to oncologists. Therapies that were once capable of destroying the tumor are rendered ineffective once one resistant cancer cell begins to multiply. Part of the challenge of developing effective cancer therapies is tackling the multiple pathways by which drug resistance arises. Isaac Israel, CEO of Kitov Pharmaceuticals, and his team are hoping to rise to this challenge. Results from recent preclinical studies suggest that their small molecule drug, NT-219, can effectively block two signaling proteins that function in two different resistance pathways.

Dance Biopharm today said it rebranded as Aerami Therapeutics and will move its headquarters to Durham, N.C. The rebranding comes as the company seeks strategic partners for its Dance 501 inhaled human insulin. The company also hopes the rebranding will help progress into pivotal registration studies and expand to include inhaled therapeutics for endocrinology diseases.

In the not very distant future, Type 2 diabetes patients may have an option to inhale insulin that acts faster and lasts longer than Lilly’s injected insulin Humalog or its generic, lispro, the current standard treatment. Durham-based Aerami Therapeutics, which changed its name from Dance Biopharm Holdings Inc. on Wednesday, reports that data from its Phase 2 clinical study of Dance 501, a novel gentle mist formulation of human insulin administered with its smart inhaler, showed it had comparable glucodynamic properties to injected insulin, but delivered a faster onset of action. It had no safety issues in the trial.

Just over 20 years ago, a Lyme disease vaccine called LYMErix was approved for sale in the United States. Researchers designed the vaccine to prevent the transmission of the tick-borne pathogen Borellia burgdorferi, which spurs a bacterial infection that can cause fever, headaches, and joint pain if left untreated.

Data from a phase 2a clinical trial of DUR-928, an endogenous, small-molecule new chemical entity, for patients with alcoholic hepatitis demonstrated that the treatment led to greater reductions from baseline in bilirubin and MELD and improved Lille scores compared with a historical control group from a University of Louisville alcoholic hepatitis study, according to a company press release.

Nurix Therapeutics is harnessing the body’s natural process for controlling protein levels to target and degrade proteins that drive cancer and other diseases. Its small molecule therapies control key enzymes responsible for protein breakdown and can be used to modulate the levels of proteins within cells. The company believes it can leverage the approach to treat a range of diseases. We spoke to Arthur Sands, CEO of Nurix, about the company’s discovery platform, its pipeline of cancer therapies, and its recent collaboration with Gilead Sciences to discover and develop drug candidates against up to five targets.