Meet a 2020 $200K Challenge Finalist: Profilagen

Although this year’s challenge will occur virtually, we want to keep the spotlight on this year’s Finalists. The PitchOff will now take place on September 17th from 5-8pm, with the Award Ceremony happening on October 1st at 5pm. Registration for the $200k Challenge will be free.

Profilagen is a start-up preclinical LLC bringing forward a technology to regenerate damaged cardiac muscle. We spoke with CEO Claudine Bruck about the inspiration behind Profilagen, and their story so far.

What was the genesis or inspiration for your idea?

In 2001, my father died of a massive heart attack at age 75—way too early, it was hard to accept.

In 2015, after an exciting and varied career in drug and vaccine development at GlaxoSmithKline, I decided to apply my experience to develop an important medicine as CEO of a startup company.

I searched local and other east coast universities for projects that fit my criteria: strong impact on public health, very innovative yet feasible, novel intellectual property, early de-risking points, and importantly, solid scientists behind it.

You cannot imagine my excitement when I finally found a project that fit all those criteria and more: We were going to prevent heart failure in heart attack patients by regenerating the damaged heart (this one is for you, Dad!). 

Leadership Team of Profilagen, a 2020 M2D2 $200K Challenge Finalist.
Claudine Bruck, Ph.D., Prof. Ed Morrisey and
Prof. Jason Burdick

We put together a team composed of extraordinary University of Pennsylvania scientists – among which the inventors of the technology – to develop a plan:

  • Professor Ed Morrisey had identified that our molecule (miR302) was able to regenerate damaged hearts by promoting the proliferation of cardiac muscle cells.
  • Professor Jason Burdick had developed a hydrogel-based technology that enabled local administration of miR302 to the cardiac muscle.
  • Professor Rob Gorman had the expertise to translate the findings from mice to pigs.

Together, they were going to show cardiac regeneration in pigs and prepare for clinical development – our expectations were sky high! Our project had the potential to end a streak of unsuccessful efforts in cardiac regeneration by replacing exogenously delivered stem cells which have shown deficiency of local implantation, by local injection of a single microRNA molecule that directs the proliferation of resident cardiac muscle cells, thus overcoming the main shortcoming of stem cell technologies.

2020 M2D2 $200K Challenge Awards Celebration Thursday Oct. 1

What are the most significant barriers you’ve faced and overcome?

Finding funding to get us started was challenging. Many VC investors saw the “very early nature of our endeavor” as a barrier.  Fortunately, we were able to start the pig work with a Small Business Innovation Research (SBIR) grant from the NIH… we are in business! 

Can you share a “Eureka!” moment—a breakthrough in the development of your idea?

Very recently, we reached our first milestone – incredibly exciting results of massive proliferation of cardiac muscle cells in injured pig hearts. We now know the dose, formulation, mode of administration, location of administration, best timing—this project IS feasible in pigs, representing a crucial de-risking step towards clinical development, so watch this space!

If you win, what’s next?

We are currently working on understanding the biodistribution and pharmacodynamic effect (i.e. miR302-induced cardiomyocyte proliferation) of our microRNA medicine in the pig heart and are nearing completion with excellent data (we are doing this with funding from a NIH-SBIR grant).  Winning the contest will allow us to initiate a control/treatment parallel group study to monitor impact on cardiac function in pigs with induced myocardial infarction, with the aim to detect a clinically meaningful impact.  This will serve as a trigger for clinical development activities.

We wish Profilagen luck in this year’s $200K Challenge!

Meet More 2020 $200K Challenge Finalists:

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