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:
Ranjan “Ron” Batra is the Chief Scientific Officer at Dyne Therapeutics, a biotech company developing therapies for people living with serious neuromuscular diseases. He joined Dyne from Lexeo Therapeutics, where he was vice president of discovery research and translation. A leading expert in RNA biology and therapeutics, he has developed cutting-edge treatments for genetic diseases. Previously, he served as a senior vice president of R&D at LocanaBio, where he advanced RNA-targeted therapies for rare disorders including Duchenne muscular dystrophy and myotonic dystrophy. His work earned him the Biocom Catalyst Award (2019), Endpoints News 20 Under 40 (2021), and recognition as one of San Diego’s Top 25 Health Care Leaders (2022). Before LocanaBio, he worked at Verily Life Sciences and conducted pioneering research at UC San Diego and the University of Florida with more than 35 publications and patents. Ron earned a PhD in genetics from the University of Florida, where he studied RNA biology and gene therapy for neuromuscular diseases.
Walk me through your career, highlighting key moments or decisions that shaped your path toward becoming a biotech CSO:
I actually started my scientific journey back in primary school, while living in New Delhi, India. I often wondered how small changes in biology—triggered by small molecules or even pills—could profoundly influence how a patient feels over the course of years or even decades.
I earned a degree in pharmacy and pursued a career in academia, focusing on RNA biology and genetic diseases. During my PhD and postdoctoral work at the University of Florida and UC San Diego, I dedicated my research to studying genetic neuromuscular and neurological disorders and developing therapeutic strategies for these devastating conditions. I became fascinated by how RNA mechanisms drive these diseases, which led me to explore emerging technologies at the time, such as RNA sequencing and RNA-targeting platforms.
A pivotal moment in my career was the founding of Locanabio. What started as a 2–3 person research concept grew into a 110-person team. We raised $160 million, built an RNA-targeted AAV gene therapy platform, and advanced it through IND-enabling studies. That experience gave me firsthand insight into venture creation, company building, leading multidisciplinary teams, navigating strategic partnerships, and cultivating lasting relationships across the biopharma and venture capital communities.
It was during this time that I truly began to understand leadership—learning from some of the most accomplished leaders in biotech, many of whom were part of the founding team at Locana. Now, as Chief Scientific Officer at Dyne Therapeutics, I’m privileged to lead research strategy and innovation in neuromuscular medicine alongside an exceptional R&D team and leadership group.
Looking back, every step—from academic curiosity to entrepreneurship to executive leadership—has been about transforming bold science into therapies that drive meaningful functional improvements for patients and their families.
At Dyne and previously at Lexeo and Locanabio, you’ve worked on rare genetic diseases. How do you approach target selection and platform optimization in such specialized indications?
I think target selection is critically important, and here’s how I—and our team—approach it. First, we start with human genetics and functional genomics to ensure there’s a causal relationship between the target and the disease. This helps reduce biological risk from the outset. Next, we evaluate whether the disease or target is amenable to our therapeutic platform or modality. That involves a deep dive into the RNA biology of the target to determine if the disease is best addressed by modulating a specific mechanism—such as RNA splicing, knockdown, or other forms of regulation.
Today, we can also apply machine learning, deep learning, and bioinformatics to support target prioritization, predict potential off-target effects, and assess safety risk—an essential component of drug development.
We also take into account both preclinical and clinical development risks. For example:
- Are there well-established preclinical models for evaluating safety and efficacy?
- Is there a clear unmet need, and can patients be recruited efficiently for a clinical trial?
- Is there regulatory precedent, or can a logical clinical endpoint be selected to demonstrate meaningful and statistically significant improvement?
Ultimately, the goal is to deliver functional benefits to patients. The ideal platform enables direct treatment of the underlying genetic cause, offers efficient delivery to affected tissues, supports redosing and durability, ensures safety, and creates a positive overall patient experience.
Dyne’s FORCE™ platform represents a promising approach to treating amenable genetic conditions in this way—providing the foundation for functional improvements that truly matter to patients.
Here at Dyne, we also place strong emphasis on early biomarker development to de-risk translational steps and strengthen our clinical path. This reflects a holistic, rigorous framework for target selection that guides everything we do.
RNA-targeted therapeutics have evolved rapidly in recent years. What scientific or technological advancements do you believe have had the biggest impact on this space?
I think the biggest advancement in recent years has been in delivery technologies. Alnylam pioneered this space with their work on GalNAc conjugates, enabling the effective delivery of oligonucleotides and siRNA to the liver. Building on that, next-generation antisense oligonucleotides (ASOs) conjugated to antibodies or antibody fragments—like Dyne’s FORCE™ platform—represent an exciting advancement. These allow for targeted delivery to extrahepatic tissues (i.e., outside the liver), enabling clinically meaningful drug concentrations that can drive functional improvements in patients.
These conjugated ASOs are now clinically validated—we have two programs in the clinic—and they are also amenable to improvements in CMC (chemistry, manufacturing, and controls) and commercial-scale production. Overall, I believe these delivery technologies are extremely promising.
We’re also seeing the use of natural AAV capsids in gene therapies, such as in the treatment of spinal muscular atrophy (SMA)—Zolgensma is a well-known example. These capsids are promising for reducing clinical doses and improving tissue targeting, which could ultimately improve safety. However, their clinical validation and large-scale commercial manufacturing are still in early stages. To reiterate, I believe GalNAc conjugates for liver delivery and antibody/antibody fragment-conjugated ASOs for extrahepatic delivery are major breakthroughs in the field.
Another key area of innovation in the oligonucleotide space is chemistry. New ASO chemistries have improved potency, safety, and delivery profiles. For instance, recent data from Biogen and Ionis on Tofersen (SALNERSEN) showed meaningful improvement over Spinraza for SMA. In just six months, the treatment achieved a 70% reduction in neurofilament light chain, a biomarker of neurodegeneration—highlighting the power of chemistry innovation.
Third, and perhaps the most widely discussed, is the advancement of computational biology and artificial intelligence. These tools have revolutionized how we study tissue and cellular health, leveraging next-generation sequencing in both healthy and disease states. They are vital for biomarker discovery and now support rational design of RNA-based therapeutics—helping to predict target accessibility, off-target effects, and optimize both manufacturing and formulation. This is crucial to ensuring that drugs are not only effective and safe but also scalable for commercial production.
You’ve taken programs from concept to IND. What are the most underrated or overlooked aspects of translational planning that early-stage companies often miss?
I’ve seen companies— and experienced firsthand—how critical it is to start thinking about translation early in the drug development process. There are several key areas that need attention from the outset:
- Platform and Target Risk – It’s essential to evaluate both platform and target-specific risks early on. This includes managing potential off-target effects. Computational tools can be especially useful here, helping to predict and assess off-target interactions—whether they impact unintended biological pathways or tissues. This de-risks development before you ever reach the clinic.
- PK/PD Modeling – Pharmacokinetic and pharmacodynamic (PK/PD) modeling is another vital component. Understanding the relationship between drug exposure and biological response, and calculating a safe and effective first-in-human dose, helps avoid surprises during clinical development.
- Immunogenicity and Safety Pharmacology – It’s important to assess the immunogenic potential of your therapeutic, especially with modalities like AAV vectors. In parallel, early safety pharmacology studies help anticipate any adverse effects that could derail clinical progress.
- Biomarker Strategy – Initiating a robust biomarker strategy early on can significantly support your ability to demonstrate clinical proof of concept. Biomarkers shouldn’t be an afterthought—they should be built into the translational plan from day one.
- Regulatory Engagement – If you’re planning to enter the U.S. or European markets, early and frequent engagement with regulatory agencies like the FDA or EMA is critical. This helps accelerate development timelines and de-risk your IND filing.
- CMC (Chemistry, Manufacturing, and Controls) – CMC is often overlooked—but it’s one of the most common reasons for IND holds or delays, especially in biologics. Building a high-quality GMP process and a well-characterized drug product early on is crucial for smooth progression into the clinic.
In summary, a strong translational plan should integrate all of these elements from the earliest stages. It should never be treated as an afterthought.
You’ve helped raise over $200M in funding across different roles. What advice would you give to other R&D leaders who are new to fundraising or investor engagement?
This is something really important to keep in mind—and I believe every scientist, especially those entering the industry, should understand this: we’re in the business of science. An effective R&D strategy should be data-driven, while also prioritizing stakeholder interests and aligning with long-term goals.
In biopharma, timelines for product approval and market launch are relatively long, so it’s critical not to lose sight of the end product. From the outset, you need a clear strategy for translating complex biology into a therapeutic vision—one that leads to real patient impact and market opportunity. If your product can provide meaningful functional improvement for patients, it inherently carries commercial value. That should be evident from day one, including in your pitch materials.
You also need to build credibility early. Investors place their trust in teams that are transparent about risk and have clear risk mitigation strategies in place. If everything were perfectly de-risked, VCs wouldn’t be investing at the early stage—they’d wait until later. So it’s okay to acknowledge risk; what matters is how you plan to manage it.
Be sure to lay out key performance indicators (KPIs) and milestones clearly—both in your research plan and in your broader business strategy. Explain how you’ll measure success and communicate progress. When selecting your indication, emphasize the unmet medical need and clearly articulate your differentiation—whether that’s through your platform, therapeutic approach, or the disease area itself. Differentiation is key to standing out in a crowded space.
Also, keep in mind: venture fundraising is a numbers game. It’s important to understand which VC firms are actively deploying capital, where they are in their fund lifecycle, and whether your pitch aligns with their investment thesis. Tailoring your pitch to each firm is essential. Don’t be discouraged by rejections—in fact, getting to a fast “no” can be valuable. Ask for feedback and use it to refine your pitch and materials. Over time, this makes your story stronger and your approach sharper.
Data always speaks louder than ideas alone. Instead of relying solely on vision, back up your story with strong preclinical data, differentiated IP, and a clear plan. That’s what makes a case truly compelling. You can also leverage partnerships. If you’ve raised early capital, securing collaborations with pharma can provide non-dilutive funding and help de-risk your platform—both of which are attractive to investors.
Finally, stay adaptable. Fundraising is rarely a linear path. Feedback from investors should inform scientific and strategic pivots. At the end of the day, fundraising is really about storytelling—grounded in scientific rigor and operational excellence.
If you had unlimited capital and talent, what unsolved problem in RNA biology or genetic medicine would you dedicate yourself to solving?
A few key areas come to mind. First, I remain deeply focused on improving the precise delivery of RNA therapeutics. While we’ve already discussed delivery as a major advancement, there’s still significant work to be done—not only in targeting specific cell types, but also in reaching precise subcellular compartments. For example, if a disease is caused by mutated RNA that accumulates in the nucleus, then the therapeutic must be delivered beyond the cytoplasm and into the nucleus to be effective. This level of precision is critical for maximizing efficacy.
There are various strategies being explored to direct oligonucleotides or siRNAs to the correct intracellular location depending on the biology of the target. I view this as a fundamental challenge in RNA biology—one where we’re making progress, but still have room for innovation.
Second, in the context of rare diseases, a single disorder can often result from multiple different mutations. While a platform might be broadly applicable, it may require different oligonucleotide products for each mutation. For example, an ASO designed to correct Mutation A may not work for Mutation B, even within the same gene—but the same delivery platform could, in theory, carry both.
However, developing separate products for each mutation becomes cost- and time-intensive, and may not be commercially viable, especially in ultra-rare disease settings. This is where regulatory innovation is needed. If we can work with agencies to establish regulatory flexibility around platform-based approaches, it may become possible to deliver customized products without requiring full clinical development for each individual mutation. I’d be particularly interested in helping streamline regulatory pathways for these types of platform-based therapies, especially in indications with high unmet need and limited commercial viability.
Lastly, I’m intrigued by the potential of trans-splicing—a platform that allows one RNA sequence to be spliced into another. This has the potential to correct many genetic diseases at the RNA level. The key challenges here are efficient delivery and optimizing the splicing reaction, both of which remain in the early stages of development. But if solved, trans-splicing could offer a powerful and versatile therapeutic approach.
These three areas—precision delivery, regulatory frameworks for mutation-specific therapies, and trans-splicing platforms—remain largely unsolved today, and I’d be excited to contribute to advancing them.
