Oncosec have developed another way of delivering genes to tumour cells without the need for viral vectors or nanoparticles. They have taken advantage of the electrotransfer method used by research laboratories to transfer DNA into cells. “When you put energy through the membrane of a cell, it causes it to become porous, and if you previously surrounded that cell with DNA plasmid, once you form those pores, the plasmids will move from outside the cell, through the membrane into the cell,” explains O’Connor.

ProQR Therapeutics has encouraging early data from a small study that show its experimental RNA-based therapy for a rare, inherited form of vision loss helped patients recover some of their sight. Based on those results, the company is planning to advance its therapy to two pivotal studies that could start later this year.

Dr. Sudarshan Hebbar, Chief Medical Officer, CalciMedica talks about developing drugs that are focused on targeting CRAC channels, calcium release activated calcium channels, in order to modulate these channels to help treat diseases that have a significant inflammatory component.  Initially developed drugs for acute pancreatitis targeting both the pancreas and the lung. Now conducting large trial in patients with severe and critical COVID-19 as many of the mechanisms that are shared in the development of lung disease with COVID-19 are shared with acute pancreatitis. Strong evidence that their drug Auxora  might be effective for a variety of disorders that cause lung injury.

That is the mission of Clene Nanomedicine, which has combined cell biology, solid state physics, and materials science to pioneer the development of Clean-Surfaced Nanocrystal (CSN) therapeutics. The company’s goal is to use this process to enhance the energetic support in neurons and change the way neurodegenerative diseases are treated. PM360 spoke with President and CEO Rob Etherington about how this science works, how these treatments differ from the current standard of care for these diseases, and what this platform may mean for other therapeutic categories in the future.

Chiasma has released phase 3 data supporting its belief that acromegaly patients will prefer to take its oral formulation over existing long-acting injectables.

How it works: The San Francisco–based company engineered a common cold virus called an adenovirus to carry instructions for making two coronavirus proteins into human cells.  There, the proteins can be made to prime the immune system to later fend off the coronavirus.

Anima specializes in treating targets long viewed as “undruggable” by targeting cellular mechanisms that regulate mRNA translation of proteins with specific biological roles in coordinating and regulating translation of individual mRNAs and pathways . . .