At least six companies have emerged to help unstick what developers say is a ‘bottleneck’ in advancing complex genetic treatments.
Developing a new drug is a long, expensive process that comes with a high risk of failure, often because would-be medicines are unsafe or ineffective.
For companies specializing in cell or gene therapies, an equally pressing concern is figuring out how to reliably make their products. Unlike small molecule or antibody drugs, genetic medicines typically involve a variety of specialized parts woven together through a complex process.
“Ex vivo,” or outside-the-body, treatments can involve a multi-week process for collecting, multiplying and modifying a patient’s cells in a laboratory. Even the simpler “in vivo” therapies have multiple pieces, including engineered viruses and synthetic genetic material, that are challenging to produce at scale.
The approvals of a dozen cell- and gene-based medicines for cancer and inherited diseases in recent years has given young drugmakers a path to pursue. But most of those approvals were won by large pharmaceutical or biotechnology companies that invested heavily in manufacturing. Startups, by contrast, don’t yet have that luxury.
Still, cell and gene therapy research is booming. More than 2,200 clinical trials testing these types of treatments were ongoing globally as of last year, according to the Alliance for Regenerative Medicine. The surge has often outstripped the capacity of large contract manufacturers, leaving startups facing waitlists that can stretch one to two years.
A growing group of new manufacturers aim to help. Since 2017, at least half a dozen companies have launched with plans to ease the “bottlenecks” slowing down aspiring cell and gene therapy developers. Many have been started by veterans of the young field and gotten the backing of top venture firms. Here’s what they aim to accomplish and how their work is progressing. READ MORE
by Gwendolyn Wu
Source: healthcaredive.com
The United Arab Emirates (UAE) Ministry of Health and Prevention (MoHAP) has established a partnership with Novo Nordisk Pharma Gulf focusing on the creation of a national scientific guide for obesity management and weight control. The collaboration also aims to enhance public awareness of cardiovascular diseases and their complications.
Pharma giant Eli Lilly is teaming up with Haya Therapeutics in a $1bn deal to find multiple regulatory-genome-derived RNA-based drug targets, as it eyes up new targets in obesity. Under the deal, the companies will use Haya’s proprietary regulatory genome discovery platform to identify and validate long non-coding RNA (lncRNA) targets for developing potential treatments for obesity and related metabolic disorders.
The sale includes custom formulation and contract manufacturing capabilities for the nutrition market from the production facilities in New Jersey and Utah in the United States, and Tamaulipas, Mexico. Financial terms of the transaction were not disclosed.
