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Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space. Maybe in 50 years’ time we’ll be using geneediting to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”.
Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.
As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. There are a growing number of biotech and pharmacompanies that have established or are establishing such end-to-end cell therapy capabilities, which can also play into how collaborations are structured in the field.
Last month we saw some movement in the pharma world, but also in some of the orbiting worlds of agencies, consultancies, and pharma investors. In addition, two team members are taking on new roles at the company, with Glen Webster named EVP of global operations and Jennifer White named global head of growth. New CEO joins UBC.
The DISC is the biggest investment AZ has ever made, and has been hailed by the pharmacompany’s chief executive Pascal Soriot as the centrepiece of what he believes could one day become a biotech hub to rival Boston and San Francisco in the US.
“In 2024, pharmacompanies and emerging biotechs will improve candidate safety profiles and reduce off-target effects by exploiting predictive intelligence unlocked by AI,” she predicts. CRISPR GeneEditing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason.
On the back of the approvals for Pfizer/BioNTech and Moderna, suddenly there is a large amount of interest from big pharmacompanies to get involved in the mRNA space. This not only allowed Pfizer to establish a COVID-19 vaccine but the company also recently announced that it had begun a study testing an mRNA flu vaccine.
For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.
MD Anderson has historically collaborated with biotechs and pharmacompanies to conduct cancer therapy research. Targeted therapies, genetic sequencing, geneediting, immunotherapies. Ochsner Health and LSU Health Shreveport have offered themselves as additional trial sites to increase patient access to studies. .
The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. To develop these novel RNA-based therapeutics, we saw a number of big pharmacompanies team up with smaller biotechs focused on developing RNA-based therapeutics.
Enzymatic DNA production company Touchlight have augmented its DNA production capabilities with a newly announced expansion to its London facilities. Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines.
GeneEditing CRISPR-Cas9 and related gene-editing technologies continue to advance, and today they are widely used to study and develop therapeutic approaches for a broad range of human diseases. As of March 18, 2024, the FDA has approved 36 gene therapies, with an additional 500 therapies in the pipeline.
Gene therapy has shown promise in treating cancers that are particularly difficult to manage such as neuroblastoma and Wilms tumor using CAR-T cell therapy, CRISPR-Cas9 geneediting and RNA-based interventions to target genetic drivers of disease.
Further, it has been observed that many pharmacompanies are directing their efforts towards introducing subcutaneous versions of their drugs by re-engineering and reformulating products that were initially developed for delivery via the intravenous route.
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