Olivia Vizier

Krystal Biotech is confident enough about its pipeline of candidates of gene therapies for treating skin diseases that has started work on a new commercial manufacturing facility. It has even given it a name.

OncoCyte president and CEO Ron (Ronnie) Andrews discussed the company’s recent acquisitions and expansion of its diagnostics menu with GEN Edge . . .

Physician, scientist and investor Patrick Soon-Shiong might be considered a “square peg in a round hole,” he admits. But the Abraxane inventor’s work to enlist natural killer, dendritic and T cells in what he calls a “triangle offense” against cancer is finally coalescing, he recently told BioWorld.

Horizon 2020, the European Union’s research and innovation programme, is entering its final year. But how has the money been used to drive pharma developments in the region? We round up the most successful and innovative projects to emerge from scheme.

This week the publishing of data on Cue Biopharma’s star immunotherapy molecule, Cue 101, revealed just how much reality there is to concept. A paper published in Clinical Cancer Research on Tuesday showed research demonstrating the ability of a biologic immunotherapy drug candidate named CUE-101 to activate tumor antigen-specific antitumor immunity.

Gene therapy developer Krystal Biotech (NASDAQ: KRYS) has appointed Jennifer Chien to the newly created role of chief commercial officer. She comes to Pittsburgh-based Krystal from the Genzyme unit of Sanofi (NYSE: SNY), where she was vice president and head of genetic diseases.

Venture capitalists are betting $20 million that technology from Immuneering Corp. will enable it to develop medicines aimed at molecular targets that have long tantalized and eluded drugmakers.

If 2019 was a breakthrough year for Watertown, Mass.-based SQZ Biotech, 2020 could be a defining year as the company tests is first antigen-presenting cell asset in the clinic to treat solid tumors that are HPV positive.

“Successful drugs targeting the Wnt pathway haven’t been developed yet because the Wnt pathway is so essential to all cells,” commented Yusuf Yazici, MD, Chief Medical Officer of Samumed. “Previous drugs in development have shown many off-target effects at effective doses, rendering them unsafe for use.”

Two decades passed between the discovery of RNA interference, a natural mechanism of gene control, and approval of the first drug built on the Nobel Prize-winning science.

Some biopharmaceutical enterprises are even specializing in specific mechanisms of action by targeting and modifying RNA through temporary and ongoing treatment to avoid safety concerns that may arise from permanent gene therapy alterations. In this article, Daniel de Boer, CEO at ProQR Therapeutics, will discuss how they are advancing RNA therapies in retinal disorders.

Aligos Therapeutics Inc., a startup headed by entrepreneurs who led venture-backed Alios BioPharma Inc. to a $1.75 billion merger with Johnson & Johnson in 2014, has raised $125 million to develop treatments for hepatitis B and other liver diseases.

As an engineering professor at Lehigh University in Pennsylvania, Richard P. Vinci has run plenty of experiments. He specializes in figuring out what makes materials break…

Forty-one-year-old David Neufeglise was a healthy family man and mechanical engineer when he started noticing constant twitching in his arm…