Surviving as a rare disease biotech during COVID-19

With trial delays, social distancing, and falls in charitable funding, the coronavirus pandemic presents some serious challenges for nonprofit orphan disease biotechs like Cure Rare Disease (CRD) – not to mention the rare disease patients in desperate need of treatments.

We spoke to the company's founder and president Rich Horgan to find out how CRD has adapted to the new environment and what situations like these can teach rare disease researchers about having a plan B.

What is CRD researching?

Cure Rare Disease (CRD) is a nonprofit that is developing custom therapeutics that are as unique to the individuals they are meant to treat. Our mission is to offer effective, life-saving treatments developed through collaborations with world-renowned researchers and clinicians, and in partnership with our generous donors – using CRISPR gene-editing technology.

I was inspired to launch CRD with the initial goal to create treatment options for my brother, Terry Horgan (age 24) who is one of 300 million rare disease patients, and currently battling Duchenne muscular dystrophy with a compromised immune system—a dangerous condition as COVID-19 continues unchecked.

What have been the biggest immediate changes to research in light of COVID-19?

For emerging biotech companies like CRD, our nonprofit whose mission is to develop customised therapeutics using CRISPR gene-editing technology for rare disease patients like Terry, the COVID-19 crisis is presenting unprecedented challenges to overcome in order to maintain continuity of medical research that is required to achieve FDA approval and ultimately dose patients in dire need of treatment options.

How has the outbreak impacted rare disease research and patients in particular?

When it comes to curing the 7,000 rare diseases that are impacting more than 300 million people in the world, time is of the essence, and pressing pause on medical research initiatives translates into lives inevitably lost, as those individuals and their families would ultimately be left behind with no treatment options.

Each rare disease may only affect a handful of people, scattered around the world, but taken together the number of people directly affected is equivalent to the population of the world’s third largest country. The outbreak is one significant additional stressor for those battling rare disease and their loved ones who may feel hopeless at this point. Moreover, with significant capital and talent being redirected to the fight against COVID-19, clinical trials and preclinical development of non-COVID-19 related therapeutics has slowed. With progressive diseases, time is of utmost urgency and is currently being lost. The slowed pace of R&D and the redeployment of resources (both public capital along with private capital) will cause a delay that will reverberate for years to come.

For patients, the battle against rare disease could be characterised as an ‘offensive defense’ strategy. This means that patients buy time through physical therapy and managing symptoms, for instance, while therapeutics are being developed.

Since the outbreak, the strategy was forced to shift into a defensive stance only, something that will inevitably fail when fighting a progressive disease. Thus, for rare disease patients, the proverbial light at the end of the tunnel continues to move further away. A halt in progress is effectively a regression in progress.

What are the biggest challenges in keeping trials going?

For clinical trials, the difficulty lies in the in-person visits for both follow up as well as therapeutic administration. If hospitals are rampant with COVID-19 patients, then rare disease patients face increased risk at contracting the virus when visiting the clinic. For this reason, many companies will postpone starting new clinical trials and delay trial visits for existing clinical trials.

Moreover, clinicians who acted as clinical investigators for clinical trials are being redeployed to manage the significant number of COVID-19 patients. With no clinical investigator, clinical trials grind to a halt. These represent significant challenges in maintaining clinical trial continuity into the indefinite future.

How have companies like yourselves had to adapt? What new ways of working have you had to implement?

Emerging biotech companies are carefully juggling new safety guidelines, revamping core business strategies, and applying for access to emergency capital to push forward and maintain the continuity needed to survive and thrive amidst an unprecedented economic downturn.

Per social distancing mandates, emerging biotech businesses are preparing for and may already be shifting all medical research from closed academic labs to exempt commercial collaborators. Many universities which regularly conduct research in partnership with biotech companies, through sponsored research agreements, have closed critical functions such as animal shipment and are asking principal investigators to end non-essential experiments.

With academic partners in lockdown, we have swiftly moved our pending studies from Yale University in New Haven to Charles River Labs in Massachusetts. Moreover, we are conducting cross-functional team meetings virtually, while ensuring that contingencies are in place should an individual critical to a study fall ill.

Our collaborators have measures in place to reduce the likelihood for transmission. Having a plan B is more essential than ever now.

How quickly have you had to make these changes?

As the situation began to grow more dire in China, we created contingency plans for our immediate studies in fear of risking delays. No one wants to use a plan B but contingencies can win the day when the unexpected happens. One thing we realised as an industry was that while the world is shaken by the medical and economic repercussions of COVID-19, emerging businesses in the biotech space will endure if immediate strategic measures can be quickly taken — driven by dedicated industry leaders who are willing to do whatever it takes to get the job done and to think creatively as new challenges, viral or otherwise, appear.

What have you had to do to make sure this transition is a smooth one?

In a time of uncertainty, collaboration now more than ever is a must-have. Collaboration that is inclusive of the essential skill sets, while maintaining financial integrity and being nimble enough to take advantage of contingency plans allows for a smooth transition. One of the strengths of cross-functional collaborations is the ability to smoothly transition from Plan A to Plan B.

In addition to collaboration, open communication has been equally critical – the ability to freely share data, findings and other relevant information with collaborators to help inform downstream development. This requires a degree of trust in collaborators and a departure from the status quo through a trajectory which should be maintained in a post-COVID-19 world. Though funding mechanisms often do not encourage collaboration, determined and passionate researchers are navigating through the blockades.

Do you foresee any further changes to how research is conducted being needed as the outbreak progresses?

To ensure continuity, nonprofits have to rethink how they raise money to maintain their work.

As a non-profit biotech, we rely heavily on in-person engagements. With the suspension of such activities, we are quickly identifying new methodologies to increase visibility and elevate brand awareness. For nonprofits like ours in particular, fundraising strategies are being revamped in real-time, as significant revenue sources have changed overnight.

With the cancellation of all pending in-person fundraiser events, which have traditionally raised hundreds of thousands of dollars that fuel our key research and development efforts, there is a new emphasis on consolidating necessary funding from corporate sources to stay on track, as individuals will be most impacted in charitable giving in a post-COVID-19 world.

The virtual realm is also an increasing outlet for fundraising, including home-based individual giving strategies, like partnerships with pro gamers and grocery stores—niche industries that are benefitting most right now.

Is there anything you think rare disease researchers have learnt from having to work in this way that they can still apply once the pandemic is over?

Absolutely. The idea of moving nimbly, cutting red-tape and developing creative solutions to allow research and development to press on are all lessons that should be internalised within R&D operations. The thoughtful utilisation of contingency plans is another critical component that can and should be continued once society returns to a new normal.

If nothing else, I hope that this crisis helps people realise that we are all in this together. Moreover, society is proving that it is possible to successfully treat a disease when the community rallies together around ending it. If we could apply the same focus, collaboration and sense of urgency to developing treatments for less well-known diseases, I believe the world would be a very different place.

About the interviewee

Rich Horgan is the Founder and President of Cure Rare Disease. He has a deep passion for Duchenne Muscular Dystrophy (DMD) and other rare diseases as his younger brother Terry is impacted by the condition. Rich’s interest in accelerating promising treatments for DMD has led him to form a collaboration with world-class researchers and clinicians to pioneer the rapid development of customised therapies for DMD and other rare diseases.