Scientific Director | tRNA Biotherapeutics

XtRNA Bio is a pioneering biotechnology company developing a proprietary tRNA therapeutic platform for underserved patients with rare genetic diseases and high unmet medical needs. XtRNA Bio is advancing a novel approach to correct translational defects at their root, from discovery through to the clinic — with a focus on meaningful, durable benefit for patients who currently have no options.

XtRNA's proprietary engineering capability enables both elevation and suppression therapy, delivered via synthetic and vectorized formats — giving us access to a broad and expanding universe of treatable diseases.

Scientific Director – tRNA Biotherapeutics

We are seeking an experienced and visionary Scientific Director to lead and shape XtRNA Bio’s scientific strategy and translational research programs.

This role sits at the interface of discovery, preclinical development, and company strategy, working closely with the CEO and broader leadership team. You will drive the advancement of our tRNA platform from proof-of-concept toward IND-enabling studies and early clinical readiness.

Key Responsibilities

Scientific Leadership & Strategy

• Define and execute the scientific vision and R&D roadmap aligned with company milestones (preclinical → IND → clinical entry)
• Lead the development of XtRNA Bio’s dual tRNA platform (elevation and suppression modalities)
• Translate cutting-edge RNA biology into therapeutic strategies with clinical relevance

Program Leadership

• Oversee lead preclinical programs in focus indications (tRNA elevation in neuromuscular diseases, particularly CMT2D, and tRNA suppression in respiratory diseases, particularly CF)
• Guide optimization of AAV-tRNA constructs, including vector design, capsid selection, and delivery strategies
• Drive development of synthetic tRNA delivery systems to the respiratory tract
• Supervise in vivo and in vitro studies, including rodent and NHP models, biomarker development, and functional endpoints
• Drive IND-enabling studies, including biodistribution, toxicology, and dose-finding

Platform Expansion

• Identify and characterize potential clinical applications of the XtRNA platform
• Further develop and optimize our delivery modalities
• Engage with relevant academic partners for PoC studies
• Develop and nurture a dedicated Scientific Advisory Board

Cross-Functional Collaboration

• Partner with teams in CMC and vector manufacturing, regulatory and clinical strategy, and intellectual property and business development
• Align scientific execution with fundraising, partnerships, and corporate milestones

Leadership

• Establish a culture of scientific excellence, rigor, and innovation
• Interface with external collaborators, KOLs, and Scientific Advisory Board members
• Build, mentor, and inspire a high-performing multidisciplinary R&D team over the long term

External Representation

• Present scientific progress to investors, partners, and scientific conferences
• Contribute to publications, patents, and company visibility

Qualifications

Education & Experience

• PhD (or equivalent) in Molecular Biology, Genetics, Neuroscience, or a related field
• 8–12+ years of experience in early-stage biotech/pharma, with progressive scientific leadership roles
• Strong expertise in RNA biology and/or translation mechanisms, gene therapy, particularly AAV-based delivery, and neuromuscular or rare genetic diseases

Technical Expertise

• Proven experience with AAV vector design, delivery, and analytics
• Experience with LNP delivery modalities
• Experience with preclinical models (rodent; NHP experience is a plus)
• Experience with molecular and cellular assays (RNA-seq, qPCR, histology, biomarkers)
• Track record advancing programs toward IND-enabling stages

Leadership & Impact

• Ability to translate early science into therapeutic development pipelines
• Ability to lead cross-functional collaborations in a startup or high-growth environment
• Ability to contribute to strategy, fundraising, and partnerships

Additional Strengths

• Entrepreneurial mindset with hands-on execution
• Strong communication and stakeholder management skills
• Experience in rare diseases or platform-based therapeutics is highly preferred

Why Join XtRNA Bio?

• Pioneer a new therapeutic modality: tRNA-based medicine
• Work on a platform with broad applicability, shifting from “one gene–one drug” to “one mechanism–many diseases.”
• Directly shape a company advancing toward first-in-human studies
• Collaborate with leading scientists and experienced biotech entrepreneurs
• Play a central role in delivering transformative therapies for patients with unmet needs

This role is designed to grow into a Chief Scientific Officer (CSO) or equivalent executive leadership position as the company scales.