The genes that enable a fish to re-grow limbs have been found to be present in humans, new research has found. This insight could help develop regenerative treatments in humans. Many species can regenerate limbs after amputation including fish, salamanders and geckos. This can be used as a mechanism to escape predators and heal from […]
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.
Dr. Eric Kmiec, Director of the Gene Editing Institute at Christiana Care Health System, spoke at BIO 2019 about meeting unmet medical needs with gene editing. We spoke to him about his work at Christiana Care Health System and CRISPR’s role in promoting better social equality in life science.
The FDA has cleared the most expensive drug yet, Novartis’ gene therapy drug Zolgensma, for introduction to the market. Zolgensma, created to combat spinal muscular atrophy, has been priced at $2.125 million, or $425,000 annually over five years.
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 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.
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.
For the first time, CRISPR-Cas9 has been combined with electronic graphene transistors to create a new handheld device which can detect specific mutations in the genome within minutes. The device can be used to quickly diagnose genetic disorders and diseases or determine the accuracy of gene-editing techniques.
A second individual has experienced sustained remission from HIV after their treatment ended, scientists from the University of Central London and Imperial College have found.
Roche is set to pay $4.3bn for the acquisition of Spark Therapeutics, a gene therapy company that will put the pharma giant ahead in one of the biggest growing markets around.
More errors occur in DNA replication during times of stress when resources are scares, scientists at the University of Toronto have found.
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.
An innovative gene therapy treatment for Parkinson’s seems to relieve symptoms by rewriting the brain, scientists have found. Following the success of the therapy study, the researchers are planning a larger trial to start at the end of 2019.
STAT news have put together a great article assessing the need for health care systems to get on top of the payment issue quickly. Give it a read!
The Novartis CAR-T cell therapy, Kymriah (tisagenlecleucel), will be made available to patients under the age of 25 via the Cancer Drugs Fund.