Moving your Cell Therapy from Concept to Product

Introduction

There are an increasing number of promising new stem Cell Therapy applications in research and development. When these potential therapies show promise, the challenge of moving these therapies from concept to commercialization can be a daunting and confusing task. Important steps need to be followed for success including finding funding, creating effective partnerships with organizations and fostering a positive regulatory process, including IND submission. If you have questions about the best way to move your product idea forward , then please join us for a special Ask the Expert session “Moving your Cell Therapy from Concept to Product.

Hosting this week’s session is Neil Littman, the Business Development Officer at the California Institute for Regenerative Medicine (CIRM): Mr. Littman is responsible for facilitating opportunities for outside investment in stem cell research in California for both CIRM-funded and non CIRM-funded programs by biopharmaceutical strategic partners, equity investors, and disease foundations.

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Question 1

Once proof of concept has been established, what recommendations do you have for seeking out funding for a small start-up company looking to move their product forward?

0 0 0 Total: 0 There are a variety of funding sources available for small start-up companies that have generated proof of concept (POC) data, as well as for those companies that have yet to do so and are seeking funds to establish POC. The available sources of financing at this stage in a company’s … Continued

Question 2

Can you explain CIRM’s role in furthering stem cell therapies?

0 0 0 Total: 0 About CIRM: CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for … Continued

Question 3

There are at least two manufacturing strategies to bring stem cell therapies to market. In first strategy, you can build the GMP facilities in small sections in each hospital to use in patients, whereas in second strategy you can produce your product in a larger cGMP facility. My question is, in your opinion which of these strategies is best suitable to scale up over the next 10 years?

0 0 0 Total: 0 The answer to this question is unequivocally the latter strategy – it is imperative that you start as early as possible using cGMP (current good manufacturing practice) that may be scaled-up and scaled-out as your process moves through clinical development. While having dispersant manufacturing areas at various hospitals may be … Continued

Question 4

On new Cell Therapy products in development, how early do you recommend beginning communications with FDA and how do you begin the discussion?

0 0 0 Total: 0 There are a multitude of opportunities to interact with the FDA during the product development cycle that should be taken advantage of, with the earliest interactions beginning at the “pre-pre-IND stage”. Pre-pre IND interactions are informal discussion, focused primarily on preclinical issues. These interactions are always handled by teleconference and … Continued

Question 5

How would you advice utilizing strategic partners to progress a product. What things do you want to look for in a strategic partner?

0 0 0 Total: 0 Often times a strategic partnership is a great way to advance your technology or product candidate. There are many forms of strategic relationships, ranging from collaborations where the strategic partner is very hands-on and involved in the development (often committing in-kind services), to others where the partner may simply help … Continued

Question 6

When it comes to IND submission, do you have any tips for a successful submission? Are there any things that you would recommend including or any preparatory work you could recommend?

0 0 0 Total: 0 The single best recommendation I have for a successful IND submission is to make sure that you have communicated with the FDA throughout the development process. This can be accomplished by communicating with the agency via a pre-pre-IND meeting, which is an informal discussion focused primarily on preclinical issues (and … Continued

Question 7

I work in an academic lab and we think that we have a good potential Cell Therapy. We want to start a small company to explore further, but would like some advice on how to interact with the University. Any thoughts?

0 0 0 Total: 0 Deciding when and if to “spin-out” a company from an academic lab largely depends on the ability to secure the intellectual property (IP) rights from the academic institution, the ability to finance the new entity, and the ability to properly manage the new entity in order to drive the technology … Continued

Question 8

When looking for funding, are there any ways that you can set yourself apart from the competition. A way to make your company or product stand above the rest?

0 0 0 Total: 0 This is typically what investors refer to as “positioning” and is the crux of a good pitch. The key items to discern are how is your technology/product different from the competition? Is your technology entirely novel, or does it built upon and improve what is already on the market? What … Continued

Question 9

What do you think is the most important factor in determining whether a product candidate becomes a successful product or goes nowhere. Assuming that the technology and product works.

0 0 0 Total: 0 I do not think there is one answer to this question – there are a variety of factors that will determine if a product is ultimately successful in the market including its positioning (i.e. what market segment or sub-segment you are targeting), competition, reimbursement, pricing as well as a host … Continued