Women in Science: Dr. Theresa Heah and her mission to ‘open new frontiers’ in medicine
OSP: Could you give me an overview of the work you do at Intergalactic Therapeutics and what the company focuses on?
Intergalactic’s mission is to overcome the limitations of viral-based gene therapy and develop a best-in-class, non-viral alternative. Our goal is to unlock the potential of non-viral gene therapy to bring lifesaving and life-changing treatments to patients. I’m excited to say that I believe we’ve cracked the code for delivery of non-viral gene therapy using our unique combination of proprietary technologies and we are rapidly moving towards this goal as we advance towards the clinic.
Specifically, our novel technology platform comprises three core pillars to bring local, non-viral gene therapy into reality and transform treatment paradigms.
First, we use a versatile programmable non-viral gene therapy technology called C3DNA, which stands for covalently closed and circular DNA. One of the key benefits of C3DNA is that it allows for the design and delivery of DNA vectors beyond the size limits of current viral delivery systems. Second, we have a precise and tunable system for local delivery of C3DNA called COMET. This in vivo electro-transfer based system enables non-viral DNA applications and is engineered to work with different types of C3DNA therapeutic payloads and target specific tissue. The third pillar is an innovative manufacturing process that leverages standard and widely available manufacturing infrastructures and enables optimal CMC scalability and economics.
In March, we presented data demonstrating for the first time that a single subretinal administration of a non-viral DNA payload encoding the complete human ABCA4 coding sequence resulted in durable, 12-month expression of human ABCA4 protein in adult porcine retinas. The expression levels achieved suggest potential therapeutic benefit in individuals living with ABCA4-related retinopathies.
Based on these findings, we plan to move our lead ABCA4 program into the clinic in 2024 and are also progressing a pipeline of programs targeting other ophthalmology indications. Overall, we’re extremely excited about the promise of our platform and its potential to expand the universe of possibilities for gene therapy in order to reverse historically untreatable diseases for patients in need.
OSP: When did your interest in science develop?
The root-cause for my interest in science goes back to when I was a very young child. I come from humble beginnings in Malaysia, and my father died suddenly when I was just five years old. He had an unexpected myocardial infarction when he was in his late 40s – and at that time in Malaysia, there was nothing that could be done to help him. I understood very early on that if there had been more scientific advances and medical innovations in place, that may have meant that my father could have been treated and may have survived. That’s when and how my interest in science first emerged, and from that early age I knew that I wanted to be a doctor.
OSP: What piqued your interest?
Losing my father at such a young age, and the idea that unlocking the power of science could help prevent other families from experiencing the same kind of loss, piqued my interest originally. From there, I think it was a combination of external and internal factors that motivated me to actively pursue my path. Even when I was young, I would tell my friends and teachers that I would become a doctor one day.
As I mentioned, we came from very humble beginnings, and it was pretty unusual for a girl of my age to express that kind of goal. Some people were very supportive and told me it was a great ambition. But others told me it was far-fetched and out of reach. In a lot of ways, it was the latter reaction that spurred me on the most. I’ve always been laser-focused on where I wanted to be and how I was going to get there, and I was determined to show people that I could and would reach my destination of becoming a doctor.
OSP: Can you tell us about your journey to where you are now?
As a physician-researcher and surgeon with a focus in ophthalmology, I have spent my career tackling challenging eye diseases. My background includes over two decades of drug development and commercial experience, leading projects from development all the way through approvals and launches focused primarily in gene therapy and ophthalmology.
As an ophthalmologist by training, I'm passionate about treating eye diseases, and I joined Intergalactic in January of this year because I was inspired and motivated by the company's transformational approach to expanding gene therapy. We want to realize non-viral gene therapy’s potential to bring profound change to patients in need of treatments and where currently no treatments exist.
OSP: Have you encountered any bumps in the road along the way?
Personally, I can say that there certainly have been bumps in the road. Overcoming the setbacks and getting to where I am today would not have been possible without the strong support of my family, friends, mentors, and sponsors who have cheered me on throughout my career and enabled me to do what I do each day. The other driving force that’s propelled me forward and motivated me to confront the inevitable challenges along this journey goes back to the reason I was drawn to science in the first place – the desire to open new frontiers in medicine to help patients with no options.
My largest professional growth was during my transition from big pharma to biotechs. Most large pharma companies have a diverse portfolio of products across multiple therapeutic areas, as well as a sophisticated infrastructure, established processes, and more financial resources. I learned a lot working in this environment about what can be achieved with a strong organizational structure, financial support, and a commercial excellence engine.
When I transitioned to biotech, there were a whole new set of challenges that had to be overcome in order to propel really important, potentially ground-breaking science forward for patients in need. For example, we had to be more nimble, financially disciplined, and communicate effectively and work collaboratively with multiple external stakeholders from investors, to regulatory agencies, to external partners and others – often with very small internal teams. So, I would say that it was during my time with both public, clinical-stage and small emerging biotech's that I encountered the most challenges and the most opportunities.
I’m proud to say, that working together with highly committed teams, we confronted these challenges head on to successfully navigate the complex road to bringing novel therapies to patients. Those successes range from closing Series A funding and bringing a three-person organization to the clinical stage, to positive FDA Advisory Committee meetings, to novel drug approvals and launches.
OSP: What do you feel most passionate about in your current role?
I have tremendous passion for our mission at Intergalactic Therapeutics and have spent my career building toward this moment. All of my previous experiences have prepared me for my role as the CEO as we take this company toward the clinical stage to unlock the potential of non-viral gene therapy and bring lifesaving and life-changing treatments to patients.
From this first-hand experience, I’m able to combine my expertise and passion for science and ophthalmology with deep knowledge about what it takes raise funds, perform with financial discipline, and build multi-billion-dollar companies with highly dedicated, high-performing teams who share a common goal of bringing transformational change to patients in need of breakthroughs.
Moreover, I understand the importance of creating value for investors and shareholders and communicating with and educating different stakeholders from investors to sell-side analysts to eyecare professionals. All are crucial in order to deliver on our mission.
And lastly, I’m passionate about working with passionate people. I feel very fortunate that I am supported by an incredible team of talented and collaborative people at Intergalactic, who are working tirelessly to advance a safer, more effective, more versatile, and more accessible solution to address ophthalmological disorders and beyond.
