Gene Therapy

BIO 2019 – An Interview with Irene Rombel, Senior Director and Head of Strategic Analysis at Janssen

With the recent conclusion of the Biotechnology Innovation Organization’s BIO 2019 event, we thought we’d talk to some of the fascinating individuals who were present to showcase their innovative ideas or technologies. Irene Rombel, Senior Director, Head of Strategic Analysis – External Innovation, Discovery, Product Development & Supply, at Janssen Research & Development, spoke at BIO 2019 about gene therapy and the next generation of biotherapeutics. We spoke to her about her thoughts on the gene therapy field, and the future for companies in that space.

New Clinical Trial Greatly Improves Genetic Rickets

A drug developed in part by Indiana University School of Medicine to alleviate the symptoms of X-linked hypophosphatemia (XLH), a disease which softens bones, has proven significantly more effective than conventional therapies.

Gene Therapy Could Heal Cells After Heart Attack

Gene therapy can make heart cells regenerate after a heart attack, King’s College London researchers have found. The study, published in Nature, stated that a small piece of genetic material called microRNA-199 delivered into a pig’s heart after myocardial infarction resulted in almost total cardiac recovery a month later.

New Cancer Pathway Opens Up New Treatment Possibilities

When the MDM2 gene acts with a specific protein found in cancer cells’ mitochondria, it can lead to cancer cell death. The study which discovered this fact, published by Mount Sinai researchers in Molecular Cell journal, could open new treatment opportunities for cancer patients in the future.

CEPI Provides $8.4 Million Grant to Fight “Disease X”

A new deal created by the Coalition for Epidemic Preparedness Innovations (CEPI) will use $8.4m to facilitate development of a vaccine platform to fight unknown pathogens. Under the deal, Imperial College London will work to create a self-amplifying RNA vaccine platform, which can then be made to rapidly develop anti-pathogen vaccines.

CRISPR Repairs DNA Even Without Donor Template

CRISPR-Cas9 can carry out precise genome editing even without the assistance of donor DNA templates, a team of scientists from Brigham and Women’s Hospital and the Broad Institute of MIT and Harvard have found.