An oligonucleotide treatment for muscular dystrophy has shown promising results in pre-clinical trials. It is estimated that the new treatment could be effective for over half of muscular dystrophy patients. Muscular dystrophy is a hereditary condition characterised by a progressive weakening and wasting of the muscles. It is caused by a malfunction in the gene […]
Dr Michelle Krishnan, Translational Medicine Leader in Rare Diseases at Roche, will by speaking at our upcoming Festival of Genomics. Her talk Clinical Development in Rare Diseases: Angelman Syndrome, From Disease Biology Insights to Genomic Medicine, will explain how to leverage disease biology insights to drive development of genomic medicines and discuss the cutting-edge clinical trial […]
NASH is present in nine to 18 million people in America alone. Yet no single drug has yet been approved for its treatment. To discuss why therapies are proving so elusive, and where efforts will be made in the future, FLG has gathered some of the most influential names in the field to discuss the problem in our latest webinar: New Approaches to the Old Hunt for NASH Therapies, held at 4pm on 11 July 2019.
Biopharma giant Abbvie has announced the acquisition of Allergan for $63 billion in cash and stock. Abbvie said the deal would be “transformational” for both companies, allowing Abbvie to diversify its business while focusing on scientific research and the company’s pipeline.
The amount of data captured by pharma companies today is fast outpacing best use for it. The ever-evolving scope of the field also means that many senior-level professionals do not fully understand the importance of getting data right in their business, or missing a potential opportunity that their rivals seize. Stemming from Front Line Genomics’ […]
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.
In potentially the first large-scale systematic analysis of thousands of cancer gene fusions, UK scientists have announced that one of the fusions could be a novel drug target for a number of cancers. CRISR editing was used to determine the most important gene fusions for cancer cell survival, before anticancer compounds were tested on them to see which might be repurposed to specifically target the fusions.
Cambridge University scientists have created the first living organism with fully synthetic DNA radically altered from its original state. The strain of E coli was given a smaller set of genetic instructions than its counterparts, proving life can continue with such a restricted code.
Takeda Pharmaceutical has announced the sizeable first sale in its proposed £10 billion-worth of assets to offset the debt it took on during its acquisition of Shire. The company announced it was divesting eye drug Xiidra to Novartis for $5.3bn total, and its TachoSil Fibrin Sealant Patch to Ethicon for $400 million in case.
Two companies with access to data from the 100,000 Genomes Project have identified patients with previously undiscovered life-threatening kidney and neurological diseases. The companies, Alexion and BioMarin, were part of Genomic England’s Discovery Forum, which grants certain industry research proposals access to data from the project after vetting.
Two separate studies have uncovered insights into why checkpoint-inhibiting immune-oncology (IO) drugs only work for a minority of patients, even when combined with other treatments. The first study uncovered a resistance mechanism within the gut microbiome, while the other relates to cancer cell-produced vesicles.
An FDA-approved drug to treat severe asthma also drastically improves the health of those individuals with hypereosinophilic syndromes (HES), rare types of chronic immune disorders. A larger placebo-controlled trial of the drug, benralizumab, will now be undertaken to confirm the results.
AstraZeneca has signed a $6.9 billion deal with Daiichi Sankyo for a single antibody-drug conjugate (ADC), trastuzumab deruxtecan.
Pharma giant Biogen and its Japanese partner Eisai have made the decision to halt two phase 3 trials of aducanumab, a drug created to slow Alzheimer’s by targeting brain-destroying beta-amyloid fragments. An independent monitoring committee decided that the drug was unlikely to benefit patients compared with a placebo.
Biogen has announced it will acquire Nightstar Therapeutics for around $800m, providing the pharma giant with a pipeline of gene therapy candidates for ophthalmology.