Sr. Director, DMPK / Clinical Pharmacology, Neurology

GondolaBio is a clinical‑stage biopharmaceutical company focused on developing breakthrough therapeutics for genetic diseases. GondolaBio is an independent sister company to BridgeBio, carved out in 2024 after securing $300M in private financing from external investors. The company leverages cutting‑edge biological research to discover, create, test, and deliver transformative medicines to treat patients with genetic diseases. Who You Are You are an experienced DMPK and clinical pharmacology scientist with specialized expertise in antisense oligonucleotide (ASO) therapeutics. You bring deep mechanistic understanding of ASO disposition, spanning intrathecal delivery, CNS distribution, tissue partitioning, and target engagement pharmacology. You are equally comfortable managing studies, the modeling workstation, and regulatory meetings, and you are energized by translating PK/PD insights into actionable dose and development decisions. Key Responsibilities ASO PK/PD and DMPK strategy: Serve as the lead DMPK/Clinical Pharmacology scientist for the company’s intrathecally delivered ASO programs. Define and execute the PK/PD strategy from nonclinical characterization through first‑in‑human and early clinical dose optimization, integrating CSF, plasma, and tissue PK data. Intrathecal delivery expertise: Drive deep scientific understanding of IT‑ASO disposition including CSF dynamics, spinal and brain distribution, rostral‑caudal gradients, and tissue accumulation. Identify and address key uncertainties in CNS PK that inform safe and efficacious dose selection and dosing interval. Nonclinical‑to‑clinical translation: Lead cross‑species PK/PD scaling to support IND‑enabling work and human dose projections. Partner closely with Translational and Discovery teams to design and interpret nonclinical PK and PD studies using relevant animal models and CSF biomarkers. Clinical pharmacology and PK/PD modeling: Develop population PK models and PK/PD frameworks for ASO programs to characterize variability, support exposure‑response analyses, and inform dose recommendations across patient subgroups (e.g., pediatric vs. adult, disease severity). Contribute to modeling deliverables for regulatory submissions. Biomarker and target engagement analytics: Collaborate with Biomarker, Translational, and Clinical teams to integrate PD endpoints (e.g., target mRNA/protein levels in CSF or accessible tissue) into PK/PD models and support Go/No‑Go decision frameworks. Preferred Education & Experience Ph.D. in Pharmacokinetics, Pharmaceutical Sciences, Pharmacology, or a closely related field; industry postdoctoral training or equivalent experience strongly preferred. 8+ years of pharmaceutical/biotech DMPK or Clinical Pharmacology experience, with substantial direct ASO/oligonucleotide experience. Demonstrated expertise in intrathecally delivered ASOs, including first‑hand experience with IT dosing paradigms, CSF PK sampling, CNS distribution, and tissue concentration analysis. Strong mechanistic understanding of ASO metabolism (nuclease degradation, metabolite profiling) and tissue accumulation relevant to CNS‑targeted programs. Proficiency in population PK and PK/PD modeling tools (NONMEM, Monolix, Phoenix/NLME) and simulation platforms. Experience supporting IND preparation and regulatory submissions with DMPK/Clinical Pharmacology content. Ability to operate independently and cross‑functionally in a lean, fast‑moving biotech environment. Nice‑to‑Haves Experience with pediatric PK/PD and dose extrapolation for rare neurological diseases. Familiarity with bioanalytical methods specific to oligonucleotides (hybridization ELISA, LC‑MS/MS for ASOs) and CSF biomarker assays. Exposure to platform technologies and multi‑program DMPK infrastructure relevant to RNA‑targeting modalities. Experience with splice‑switching, RNase H‑mediated, or other ASO mechanisms as contextual expertise. Track record of publishing DMPK or clinical pharmacology findings in the oligonucleotide field. Core Attributes of Successful Team Members Patient Champions who put patients first, uphold strict ethical standards, and anchor decisions in clinical and scientific integrity. Entrepreneurial Operators who take ownership and drive toward practical solutions, while exercising sound judgment, respecting established processes, and aligning with teammates and leadership. Truth Seekers who are rigorous, detail‑oriented, and intellectually honest—comfortable challenging assumptions, engaging with data, and updating their views based on evidence. Collaborative Thinkers who work effectively in data‑driven, interdisciplinary environments; listen actively; and welcome questions, debate, and constructive challenge as essential to better decision‑making. Individuals Who Inspire Excellence in themselves and those around them. High‑quality Executors who deliver against goals and milestones with precision, accountability, and appropriate urgency—without compromising scientific rigor or team alignment. What We Offer Patient Days, where we are fortunate to hear directly from individuals living with the conditions we are seeking to impact throughout the year and learn how we can improve our efforts. A culture inspired by our values: put patients first, think independently, be radically transparent, every minute counts, and let the science speak. An unyielding commitment to always putting patients first. A decentralized model that enables our program teams to focus on advancing science and helping patients. Our affiliate structure is designed to eliminate bureaucracy and put decision‑making power in the hands of those closest to the science. A place where you own the vision—both for your program and your own career path. A collaborative, fast‑paced, data‑driven environment where we inspire ourselves and each other to always perform at the top of our game. Access to learning and development resources to help you get in the best professional shape of your life. Robust and market‑competitive compensation & benefits package (Base, Performance Bonus, Equity, health, welfare & retirement programs). Flexible PTO. Rapid career advancement for strong performers. Potential ability to work on multiple GondolaBio Pharma programs across multiple therapeutic areas over time. Commitment to Diversity, Equity & Inclusion. The base pay range for this position is $285,000 to $340,000 per year. The actual compensation offered will be determined based on a number of job‑related factors, including experience, skills, qualifications, and location. A bonus plan and equity will also be offered. GondolaBio ServiceCo is an equal opportunity employer. All applicants will be considered for employment without attention to race, color, religion, sex, sexual orientation, gender identity, national origin, veteran or disability status. Employees of BridgeBio and all affiliated companies should work through their managers to inquire about and learn more about this role. #J-18808-Ljbffr GondolaBio