Senior Scientist – Expert in RNA Biology and Rare Neurodevelopmental Diseases

Role Summary

Senior Scientist – Expert in RNA Biology and Rare Neurodevelopmental Diseases based in Cambridge, MA. The role focuses on discovering and validating novel RNA-based therapeutic targets to advance our rare disease pipeline, particularly for neurodevelopmental indications. You will lead cross-functional collaboration to translate scientific insights into new programs and help expand the therapeutic portfolio.

Responsibilities

• Drive new program initiation by applying RNA biology expertise and RNA associated cutting-edge technologies (in silico prediction of RNA expression/splicing/degradation/export/structure, single nuc/single cell and bulk RNA-seq analysis, splicing analysis from RNA-seq data, in vitro high-throughput splicing assays), for rare neurodevelopmental diseases
• Design, develop and conduct in silico, in vitro and in vivo mechanism-of-action, and proof-of-concept studies to validate targets and evaluate therapeutic strategies
• Contribute to interdisciplinary research programs in a highly collaborative manner across internal groups and with external partners, with opportunity to grow into a project leadership role
• Prepare high-quality analyses, interpretation, documentation, and presentations to support project milestones, publications, patent filings, and potentially regulatory (IND/CTA) filings
• Present findings and progress in internal meetings and external venues as appropriate (e.g., publications, conferences, collaboration and governance meetings)

Qualifications

• Required: Ph.D. or equivalent advanced degree in Molecular Biology, Cellular Biology, In vivo Biology or a related discipline, with 3+ years of relevant experience in a biopharmaceutical or academic research setting
• Required: Deep expertise in neurodevelopmental diseases biology and in vivo and in vitro models
• Required: Expertise in RNA homeostasis including RNA regulation and processing (e.g., splicing, stability, transport), the roles of regulatory RNAs and translation regulation mechanism
• Required: Demonstrated ability to produce high-quality scientific documentation, including experimental protocols, standard operating procedures (SOPs), technical reports, and presentations
• Required: Proven track record of meaningful contributions to research programs, evidenced by scientific publications and/or presentations

Skills

• Ability to work independently and collaboratively within cross-functional, matrixed teams
• Highly adaptable, with strong multitasking skills and the ability to manage shifting priorities and timelines
• Strong analytical and problem-solving skills with sound scientific judgment
• Clear written and verbal communication skills, with the ability to convey complex concepts clearly
• Interpersonal strengths that foster effective collaboration and the ability to build productive working relationships
• Outstanding organizational skills, attention to detail, and commitment to maintaining rigorous documentation

Additional Requirements

• Preferred: Hands-on experience with disease-relevant in vivo models, including functional and phenotypic assays such as EEG, PPF, SYNPLA, fEPSPs, synaptic plasticity measurement techniques, behavior, cognitive and motor functional assays
• Preferred: In vitro skills in iPSC culture, imaging microscopy, high-throughput imaging microscopy, in vitro neuronal activity (MEA), calcium imaging
• Preferred: Molecular skills in gene expression analysis (qPCR, ddPCR, WB, ELISA/MSD), molecular cloning, vector design, gene editing (e.g., CRISPR)
• Preferred: Experience with discovery, optimization, and development of small molecules and/or oligonucleotides
• Preferred: Familiarity with drug discovery and development processes