At least six companies have emerged to help unstick what developers say is a “bottleneck” in advancing complex genetic treatments.
Editor’s note: BioPharma Dive, as part of Emerging Biotech Weekly, is taking a closer look at competitive areas of startup activity. We aim to give an overview of the companies developing new technology and their goals.
Developing a new drug is a long, expensive process with a high risk of failure, often because would-be medicines are unsafe or ineffective.
A pressing concern for companies specializing in cell or gene therapies is how to make their products reliably. Unlike small molecule or antibody drugs, genetic medicines typically involve various 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.
In recent years, the approvals of a dozen cell- and gene-based medicines for cancer and inherited diseases have given young drugmakers a path to pursue. But large pharmaceutical or biotechnology companies that invested heavily in manufacturing won most of those approvals. 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 aims 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 have gotten the backing of top venture firms. Here’s what they aim to accomplish and how their work is progressing:
What are the main bottlenecks in cell and gene therapy manufacturing?
Cell and gene therapies involve materials not used in many other products the pharma industry is well-versed in producing.
Scientists design synthetic genetic material to deliver to patients, either via their cells, benign viruses known as vectors, or specially made bubbles of fat. Constructing these treatments is tricky even in a research setting, where small amounts of such material might be required for early experiments. But it’s much harder for companies running clinical trials or preparing for mass production.
Manufacturing delays can wreak havoc on young companies, causing them to miss milestones that could endanger future funding. Established gene therapy biotechs like UniQure or BioMarin Pharmaceuticals have spent years and millions of dollars to build their plants. But startups and academic labs — where many approved cell and gene therapies originated — can’t afford that.
“Academics have truly cutting-edge research, and I have been blown away by some of the creative ideas, novel modalities, and breakthrough innovations that came about,” said Ran Zheng, the CEO of Landmark Bio. This Massachusetts-based company caters to cell and gene therapy developers. “But that information needs to be translated into therapeutics, and this is the biggest, and probably the first, hurdle [startups] have to overcome.”
Turning to contract manufacturers like Thermo Fisher and Catalent can be a solution, but it brings problems. Transferring technology from a small lab to a larger organization can be arduous and require troubleshooting for glitches that arise in the process.
Big CDMOs may also prioritize more lucrative work with larger biotech and pharmaceutical firms. And they’re struggling to meet the surging demand for cell and gene therapy manufacturing tools.
Building up capabilities internally can be costly for startups. Viral vectors, for instance, are expensive to make and handle.
“You often see companies trying to own their manufacturing, and unfortunately, in this environment, if the product’s not successful, that’s a heavy capital and operating expense to carry,” said Mike Paglia, a senior executive with ElevateBio. This richly funded startup helps manufacture cell and gene therapies.
Reference: https://www.biopharmadive.com/news/cell-gene-therapy-manufacturing-cdmo-biotech-startups/653275/
If you enjoyed this article, please let us know.