Welcome to Partnology’s Biotech Leader Spotlight Series, where we highlight the remarkable accomplishments and visionary leadership of biotech industry pioneers. This series is about showcasing the groundbreaking strides made by exceptional leaders who have transformed scientific possibilities into tangible realities. Through insightful interviews, we invite you to join us in following the inspiring journeys of these executives who continue to shape the landscape of the biotech industry. This week we are recognizing:
Alan H. Cohen, MD is the Chief Medical Officer at Arcturus Therapeutics. He is a board-certified Pediatric Pulmonologist with a longstanding interest in cardio-respiratory illnesses. He has been clinically active for the balance of his 35+ years of practice, with a focus on caring for people with Cystic Fibrosis, as well as previously co-directing the largest and most active pediatric lung transplantation program in North America. In addition to his many years of clinical practice, biomedical and basic science research, and teaching, he has dedicated over 25 of his 35+ year-long professional career to drug discovery, in the Biotech & Pharma clinical/medical research space, at both small, entrepreneurial pre-IPO start-ups, as well as more well-established public companies worldwide. He has most recently worked in the fields of heart failure (amyloid-related) and pulmonary hypertension, respiratory infectious diseases (P. aeruginosa, influenza and RSV), as well as gene editing/gene therapy targeting monogenetically driven conditions, including rare and more common diseases such as alpha-1 antitrypsin deficiency, amyloidosis, cystic fibrosis, hemophilia and Fabry Disease.
His research and drug development career have included roles of increasing responsibility and leadership at companies such as: 4D Molecular Therapeutics (SVP/TAH Pulmonary, CV & Rare Diseases), Metagenomi (SVP/CMO Clin Dev Gene Editing), Eidos/BridgeBio (VP), Bayer (GCL), Aridis Pharma (SVP), Therabron Therapeutics (SVP/CMO), Eddingpharm (CMO), Boehringer Ingelheim, InterMune (SVP – acquired by Roche / Genentech), MAP Pharma (VP – acquired by Allergan), Jazz Pharma (CMO/VP) & Medimmune (acquired Astra Zeneca). Dr. Cohen has remained clinically active throughout his professional medical career, including direct patient care as well as teaching medical students, residents and fellows, most recently serving as adjunct clinical and teaching faculty at Stanford University School of Medicine – in the Dept. of Pediatric Pulmonology.
Arcturus has built a differentiated position around self-amplifying mRNA and its LUNAR® delivery platform—where do you see the greatest clinical advantage of this approach relative to first-generation mRNA therapies?
The best way to answer it is by looking at Arcturus Therapeutics’ experience with our vaccine development platform—most notably our COVID-19 vaccine program utilizing self-amplifying (sa) mRNA, as well as our LUNAR® lipid nanoparticle (LNP) delivery platform.
Traditional mRNA vaccines help protect against infectious diseases like COVID-19 and influenza by providing a blueprint for cells to produce a protein that trains the immune system to recognize and fight viral pathogens. In contrast, self-amplifying mRNA (saRNA) vaccines instruct the body to generate significantly more mRNA and protein, thereby enhancing the immune response.
As a result of this pioneering work, Arcturus has evolved into a global mRNA medicines and vaccines company with several enabling technologies. These include our LUNAR® lipid-mediated delivery platform, our STARR® /STAR mRNA technology, and our integrated drug substance and drug product manufacturing capabilities.
Together, these advancements enabled the development of ARCT-154 (KOSTAIVE®), the first self-amplifying mRNA COVID-19 vaccine approved for safe and effective use in disease prevention.
While first-generation mRNA therapies and vaccines have already demonstrated remarkable speed and efficiency in vaccine and genetic medicine development, the next step is to further enhance these technologies to achieve even greater efficacy. That’s where Arcturus is focused today—advancing self-amplifying mRNA and continuing to refine lipid nanoparticle delivery systems to unlock the full potential of this platform.
With programs in OTC deficiency and cystic fibrosis, what are the unique considerations when applying mRNA technologies to rare genetic diseases versus infectious disease vaccines?
The COVID-19 pandemic was both a blessing and a curse. It brought mRNA vaccine technology to the forefront and, in doing so, undoubtedly saved millions of lives worldwide.
The next frontier is applying these genetic medicines as therapies for a broad range of diseases—particularly rare, genetically driven conditions. I’m old enough to remember the Human Genome Project in the 1990s, which provided a foundational map of the human genome. That work has since given us a much clearer roadmap for hundreds of rare diseases that still have little to no effective treatment options.
Advances in mRNA technology and lipid nanoparticle (LNP) delivery systems now enable the administration of mRNA encoding therapeutic proteins, allowing for in vivo production of proteins that are missing or dysfunctional in rare disease patients. In that sense, mRNA biotherapeutics represent a natural and highly promising platform for making meaningful progress in rare disease drug discovery.
Another key advantage is scalability—mRNA platforms can leverage a relatively standardized production pipeline, which has the potential to streamline development across multiple programs.
That said, there are significant challenges. Unlike vaccines, which are typically systemic, therapeutic applications require precise, organ-specific targeting. For example, delivering mRNA to the lungs for cystic fibrosis or to the liver for metabolic disorders—such as OTC deficiency in urea cycle disorders—requires fundamentally different approaches. Each indication demands its own tailored delivery strategy.
Ensuring that sufficient mRNA reaches the right tissue, in the right amount, to enable proper in vivo protein expression and physiological function is a complex problem. It requires careful design, optimization, and execution.
These are just a few of the challenges we face as we work to develop novel treatments—and ultimately potential cures—for the many rare diseases that still carry significant unmet medical need.
How are regulators evolving in their expectations for newer modalities like self-amplifying mRNA, and where do you still see the greatest ambiguity or risk?
Hardly a week goes by without seeing the U.S. Food and Drug Administration and other regulatory bodies in the news. It’s clear that regulators are moving toward a more flexible, indication-driven framework for saRNA. At the same time, the technology’s amplification mechanism, lower-dose requirements, and broader therapeutic potential are pushing drug development into truly novel territory.
As a result, scientists and developers need to strike a careful balance between rapid innovation and rigorous standards—ensuring robust preclinical safety data, well-defined manufacturing controls, and proactive engagement with regulators to navigate evolving expectations.
In short, it’s a moving target. But it’s also an incredibly exciting time to be working in novel drug development, especially in these uncharted areas, where the potential for meaningful advancement feels almost limitless.
You’ve led programs from Phase 1 through commercialization—what separates companies that execute well in clinical development from those that struggle, especially in today’s capital-constrained environment?
We’re certainly familiar with the challenges that come with that. Biotech companies operate in a high-stakes, resource-intensive environment where execution quality and clinical development ultimately determine survival and success.
In my view, the most successful companies build multifunctional clinical development plans (CDPs) that integrate strategy, regulatory clarity, statistical rigor, and operational feasibility in a finely tuned balance. These plans aren’t static—they require continuous updates as the therapeutic and competitive landscape evolves. Equally important is maintaining a patient-centric approach and working closely with the care community throughout the entire development process.
There are a few core tenets that tend to separate the most successful companies from the rest:
- Rigorous, holistic clinical development planning
- Agile, cross-functional execution with early identification of risks and red flags
- Optimized use of internal and external resources, including CROs and key opinion leaders
- Patient-centric, adaptive trial designs
- Early, robust, and integrated CMC strategies
- Leveraging AI and automation for data-driven decision-making
- A strong culture of strategic foresight, operational discipline, and continuous learning
By contrast, less successful biotechs often struggle with inadequate planning, poor cross-team coordination, misalignment with regulators, gaps in CMC and manufacturing expertise, suboptimal trial design, and rigid or resource-constrained execution.
More often than not, the difference isn’t scientific innovation—it’s precision in execution. Biotech success is ultimately a complex interplay of planning, execution, resourcing, and the ability to adapt and learn over time.
Having led development across biologics, gene therapy, and now mRNA, what have you had to unlearn or rethink when it comes to moving from scientific discovery to clinical execution?
I sometimes joke that at this point I’ve probably forgotten more than many people learn in a lifetime. But when you train in science or medicine, you’re really signing up for a career of continuous learning and adaptation—that’s part of the deal. And honestly, that’s been one of the most rewarding aspects of the second half of my career, as I transitioned from academic medicine and patient care into drug development across a range of biotech and pharma companies, both large and small.
Moving across modalities—from small molecules and drug-device combination therapies to biologics, gene therapy, and now mRNA—has required me to unlearn long-held assumptions and rethink how we translate science into viable, scalable clinical programs.
Take gene therapy versus mRNA, for example. Gene therapy often aims for a one-time, potentially curative intervention through durable or permanent expression. mRNA, by contrast, is transient—its protein expression is rapid, potent, and short-lived. That fundamentally changes how you approach dosing, efficacy endpoints, and long-term follow-up. You shift from a “one-and-done” mindset to thinking in terms of repeat or pulse dosing, while also considering immune memory and tolerance.
There are also important differences in mechanism and safety. Gene therapies may involve genomic integration or long-term expression of transgenes, whereas mRNA avoids genomic integration, reducing certain long-term risks—but also requiring you to design around non-persistent expression. As a result, manufacturing priorities shift: stability, delivery efficiency, and tissue targeting become critical, as does planning for repeat dosing and rapid clearance.
mRNA’s ease of design and scalability were game changers during COVID-19, enabling companies to move incredibly quickly from concept to clinic and ultimately to patients. But that speed also changes expectations—you can’t rely on the same slow, bespoke development model often used in biologics. Instead, you need to integrate manufacturing readiness early, from nucleic acid synthesis through lipid nanoparticle formulation, and align that with accelerated regulatory pathways.
Another key shift is moving from a single-asset mindset to a platform mindset. mRNA’s modularity allows it to be adapted across infectious diseases, oncology, and monogenic disorders. That means designing systems that can rapidly swap sequences, maintain consistent quality, and support regulatory continuity across multiple indications.
That’s very much the approach we’re taking at Arcturus Therapeutics here in San Diego.
In essence, I’ve had to move away from the idea that a single, permanent fix is always the goal. Instead, it’s about balancing speed with rigor, integrating development and manufacturing from the outset, and building platforms that can adapt quickly. It’s not just a technical shift—it’s a philosophical one, in how you define success, measure outcomes, and ultimately bring therapies to patients.
If you were advising a first-time biotech CMO today, what are the 2–3 priorities you’d tell them to get right in their first 12 months?
For a first-time biotech CMO—and I’ve had the good fortune of serving as CMO at eight companies—I think the first 12 months are all about building credibility, establishing the right support systems, and aligning clinical strategy with business goals.
Early on, it’s critical to assemble a strong foundation: a dedicated clinical operations team to manage trial logistics and compliance, regulatory experts to guide agency interactions and submissions, and medical affairs professionals to engage key opinion leaders and build external credibility. Having a trusted colleague or direct report with deep experience in medical monitoring, data management, and clinical operations can free you up to focus on high-level strategy.
You also need to quickly understand your company’s culture, adapt to it, and demonstrate competence right away. One of the most effective ways to do that is by prioritizing one or two well-planned, highly visible clinical milestones—such as launching a pivotal trial or advancing a key regulatory submission. Delivering on those early builds trust with cross-functional teams, investors, and regulators, and positions you as a decisive leader.
A successful CMO also has to balance patient-centric science with commercial strategy. In that first year, it’s essential to work closely with the CEO and broader C-suite to ensure the clinical development plan supports market access, investor confidence, and long-term growth. That means translating scientific priorities into clear, evidence-based milestones that resonate with both internal and external stakeholders.
At the same time, trust your judgment—it got you here. But never forget that drug development is a team sport. We build on the work of those around us, and success depends on the collective effort of incredibly talented teams. It’s important to recognize contributions, advocate for your team, and plan for their success alongside your own. And, as my parents always told me, be kind—it goes a long way.
Most importantly, never lose sight of why you’re doing this: patients. The therapies we develop can be life-changing, even life-saving. Staying connected to that purpose keeps you grounded and focused. That’s why I’ve always encouraged clinician-scientists on my teams to remain connected to patient care whenever possible—even if it’s occasional or pro bono work.
Personally, I stayed clinically active at Stanford University through the pandemic. More recently, I’ve been less connected due to time and geography, and I genuinely miss it. That connection to patients is a powerful reminder of what’s at stake—and why the work matters.
