Senior Scientist – Expert in RNA Biology and Rare Neurodevelopmental Diseases

Job Title: Senior Scientist – Expert in RNA Biology and Rare Neurodevelopmental Diseases

Location: Cambridge, MA

About the Job

Are you ready to shape the future of medicine? The race is on to speed up drug discovery and development to find answers for patients and their families. Your skills could be critical in helping our teams accelerate progress. 

We are seeking a highly motivated and skilled scientist with deep expertise in RNA biology combined with neurodevelopmental diseases. The ideal candidate will bring deep knowledge and robust hands-on experience in RNA splicing regulation, miRNA and long non-coding RNA processing, RNA degradation mechanisms as well as perform state-of-the-art computational and in vitro techniques associated to prediction, analysis and validation of those RNA processes. The candidate would have solid knowledge and experience working on neurodevelopmental diseases such as Fragile X syndrome, Dravet syndrome, Angelman syndrome or SYNGAP1 syndrome. The candidate will have hands-on experience in vitro and in vivo skills to support phenotypical assessment of neurodevelopmental disease mouse models as well as to lead exploration of novel pathways and targets. We are a team that encourages creativity and proactiveness to unlock new therapeutic avenues. This is an exciting opportunity to make an early and meaningful impact on therapies for patients with devastating neurodevelopmental diseases. 

In this role, you will lead efforts to discover and validate novel RNA-based therapeutic targets and strategies. Your work will play a critical role in advancing our rare disease pipeline, particularly in rare neurodevelopmental indications. You will collaborate closely with cross-functional teams to translate scientific insights into new programs, contributing to the strategic expansion of our therapeutic portfolio.

We are an innovative global healthcare company with one purpose: to chase the miracles of science to improve people’s lives. We’re also a company where you can flourish and grow your career, with countless opportunities to explore, make connections with people, and stretch the limits of what you thought was possible. Ready to get started?

Main 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)

About You

Basic Qualifications:

• 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

• Deep expertise in neurodevelopmental diseases biology and in vivo and in vitro models

• Expertise in RNA homeostasis including RNA regulation and processing (e.g., splicing, stability, transport), the roles of regulatory RNAs and translation regulation mechanism

• Demonstrated ability to produce high-quality scientific documentation, including experimental protocols, standard operating procedures (SOPs), technical reports, and presentations

• Proven track record of meaningful contributions to research programs, evidenced by scientific publications and/or presentations

Preferred Qualifications:

• Hands-on experience with disease-relevant in vivo models, including functional and phenotypic assays such as Electroencephalogram (EEG), Paired-Pulse Facilitation assay (PPF), SYNaptic Proximity Ligation Assay (SYNPLA), field excitatory postsynaptic potentials (f