In our third installment of our Regenerative Medicine panel, our experts discuss their views on potential catalysts and disruptors for cell and gene therapies.  Below is a condensed and edited version of Part 3 of a panel discussion moderated by Kevin Hampton, Chief Commercial Officer at Kineticos.


  • Susan Nichols – CEO, Falcon Therapeutics
  • Flagg Flanagan – CEO & Chairman, DiscGenics 
  • Bob Hutchens – CEO, StemBioSys

Kineticos: When we’re evaluating emerging markets at Kineticos, we look at what we call catalysts and disruptors. It’s especially important to know about all of the advanced science because any threat to current standard of care could impact development strategies. Knowing what you know today, are there any potential catalysts out there that could really allow for a step change in Regenerative Medicine similar to what we’ve seen with how checkpoint inhibitors changed the  oncology landscape? On the flip side, what are any potential disruptors that could put a strain on regenerative medicine?

Susan: I’m starting to see the manufacturing enhancements that were promised in the past, come to market with unique capabilities and efficiencies. Before, closed automated systems were a dream and they are now starting to become a reality in increments. I also see the beginnings of hospital-based therapy manufacturing as well. We’re making strides at eliminating complex logistics so we can deliver a therapy to the patient at the earliest time point. It is not exactly point-of-care but it’s closing that gap between the patient’s diagnosis and their option to receive a cell or gene therapy. We are also seeing some steps to cost down these therapies that would allow them to become more mainstream such as alternatives to viral vectors. We need to get to a point where it is more cost-effective for both patients and the insurance company.

Kineticos: Very recently, Bluebird had to push back the launch date of their newly approved gene therapy due to manufacturing issues. If we could solve some of those challenges, that will only help us getting these therapies to the patients quicker.

Susan: We are solving some of these challenges much earlier now with companies coming up behind Bluebird. Not too long ago, the industry was looking at closing and automating around Phase 2. Now, companies are planning and implementing manufacturing enhancements pre-IND and developing robust scale-up manufacturing plans that are clinical trial phase appropriate.

Bob: Thinking about cell therapy, I have always maintained that if we can get a few big wins, or even one big win, we will see the FDA’s and investors’ attention change. Think about something as prevalent as type 2 diabetes – if we could cure that using stem cell treatments, that would be incredibly profound in terms of the effect on patients. We have seen CAR-T make a significant impact on certain cancers and as that starts to progress, the stem cell world and pure regenerative medicines will get moving.

In terms of barriers or disruptors that could cause a strain on all of this, I worry about the industry getting tainted. There are stem cell clinics out there performing treatments that violate the spirit, if not the letter of FDA regulations, and that could harm the entire industry. For example, there was a case not too long ago where a federal judge made a ruling about manipulated cells. This is just one example of the kinds of players who could harm the entire industry.

Flagg: In terms of barriers or disruptors, we see quite a few companies in the musculoskeletal space that are abusing the 361 designation for minimally manipulated products by either misrepresenting the nature of their products, or by making unsubstantiated efficacy claims about their products without having undergone thorough evaluation in an FDA-allowed clinical trial. On the other hand, DiscGenics and other cell therapy companies like ours that are pursuing a BLA through an IND for a 351 manipulated product are required to prove not only that our products have a therapeutic effect, but also that they meet critical safety standards and are manufactured consistently from lot to lot. The regulators are working hard to ensure that the distinctions between the two designations are made clear and that those who must adhere to the rigorous regulatory standards established by FDA are doing so. However, if we’re going to be successful in bringing safe and efficacious products to patients, we must continue to differentiate ourselves from the bad actors in the space.

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