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Advice for Starting A Rare Disease Company

NOVEMBER 12, 2014

I never imagined I would be the CEO of a small rare disease company.  I am a scientist who came up through the big pharma, biotech, and consulting route of scientific career progression. In fact, I have not been in this position for long, I may not be the best at it and who knows how much longer I will be doing it after this post!  However, I am going to give it my all though. Getting here required good networking, a lot of writing,  and tenacity (common skills that many of you likely possess). And I work with some of the most amazing people, just like you!

Our company’s goal is simple and that is to collaborate with the best scientists we can find to help bring treatments to the clinic for one of the 7000 rare diseases. These treatments are urgently needed so we can literally save people’s lives. In the past few weeks with the realization that there are many more diseases that need help, I’ve even started a second rare disease company. But before I discuss that one, I need to briefly go back in time three years to explain my present situation and how one meeting changed my career direction, before I was a CEO.

My First Encounter with A Rare Disease Parent-Advocate

In November 2011, I presented at the Partnering For Cures conference on the Tuberculosis research I had been involved in for a few years while with a software company. I had also recently published an article on in silico repositioning of approved drugs for rare and neglected diseases that had caught the attention of at least one attendee. One of the very useful aspects of this meeting is the opportunity to set up small meeting with attendees. The first meeting I had was with a rare disease parent advocate named Jill Wood. We had never met before, but she told me what had happened to her family over the past few years. Jill explained how her 3 year old son Jonah was diagnosed with a rare disease called Sanfilippo Syndrome, (a specific type called IIIC). She told me how she had searched through the internet looking for scientists and clinicians to consult, and how she and her husband Jeremy had founded their own foundation called Jonah’s Just Begun and had put together a scientific advisory board. Jill had raised some money which went towards funding a knock out mouse for the disease and work with Alexey Pshezhetsky in Montreal. She wanted advice on what to do next. I was a little dumbfounded. Her son was the same age as my daughter, what would I do in her shoes? I suggested “why don’t you start a company, because then you can go after small business grants at the NIH”.

After the meeting, Jill set up the company Phoenix Nest and we worked with Alexey to put together an STTR that was submitted in April 2012. In the interim, I worked with Alex Clark to make a mobile app focused on rare diseases. During the year Jill introduced me to another rare disease parent advocate Lori Sames of Hannah’s Hope Fund, who had a company called BioGAN and wanted to write a grant with Allison Moore of the Hereditary Neuropathy Foundation. So I helped put together a grant for Charcot-Marie-Tooth Disease that was submitted that August. In the intervening period the Sanfilippo grant proposal did not get funded so we resubmitted the proposal later in the year, as it appeared reviewers had an issue with funding research on Sanfilippo syndrome IIIC work being done in Canada. Ultimately none of these proposals were funded but they provided the ground work for other proposals and a paper in 2013. In addition, all three foundations submitted and won prizes offered by the Rare Genomics Institute and Assay Depot later in 2012. After volunteering with these groups the collaborations ultimately led to spending a part of my time doing more consulting with each group. In late 2013 I helped prepare a massive RDCRN grant proposal with Lori, Allison, and a colleague Renee Arnold. This was about the equivalent of submitting 6 small business grants simultaneously. Unfortunately, it too was not funded but we were able to condense it and write a commentary on research we think needs support for these rare diseases. Going through this process helped build connections at the NIH such that it has also spawned additional grant proposals with other collaborators.

Later in 2013 Phoenix Nest submitted another STTR with Patti Dickson at LA BioMed. This (after what seemed a very long time) was ultimately funded at the first attempt a few months ago . Phoenix Nest was indeed a real company and Jill became CFO and made me the CEO. We now had to find a postdoc, sign contracts and manage a budget. We also had finally got to the point imagined two and half years earlier. In the interim we have also been looking at other licensing opportunities and submitting other grants with Patti and Alexey. We are off and running.

Advice from the CEO

After this 3 year journey it is probably a good point to offer some advice. Anyone can start a company. In order to start a rare disease company, parents, patients or advocates that have started their own foundation and know all the science and scientists are in a special position. They almost have a “superpower”, they can approach prominent scientists, company leaders, regulators, celebrities and politicians and they will generally listen to them. They have most of the skills and knowledge, which when combined with good legal and accounting advice could form the foundation of a strong company. The other key ingredient is to find one or more scientist with whom you can submit grants and ultimately license what comes out of the work. Having someone involved that can write grants and papers for the company, I would humbly say is an asset, because it represents an alternative to fund raising and there are challenges to publishing as most journals only accepted articles from scientists.

The ability to also never take no for an answer or give up under the most trying of circumstances are important characteristics to consider. Rarely will you win a grant on the first attempt without truly exceptional science and ideas for a product. In the case of Phoenix Nest, we had submitted several different proposals before winning a grant. Even rarer, a potential treatment for your disease is unlikely to land in your lap. Rare disease parent /advocates can really move mountains and therefore starting a company is just an alternative way to get to the endpoint of finding treatments for the disease they are fighting. It is literally a marathon, so pacing yourself, surrounding yourself with good people that know how to develop drugs and not getting overwhelmed by it all are critically important.

Advice to Myself

So it is at this point I have decided to take my own advice, while I am not a rare disease parent or patient, I have seen what they can do and I want to help more of them do the seemingly impossible. What if we could form a company to try to use this approach and scale it to many other rare diseases? I hope in future posts to document the development of this new company which has just been incorporated and how this fits into my other full time work. Please watch this space as my journey continues because this is hopefully a start towards a bigger goal.

Advice to Scientists

If there are scientists reading this and you get the opportunity to meet a rare disease parent, patient or advocate please listen to them because they will be hanging on every word you say. Who knows one of them just might go off and start a company. The rest they say is history.



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