Senior Scientist –Molecular, Cellular and In vivo Expert in Rare Neurodevelopmental Disorders

Job Title: Sr. Scientist –Molecular, Cellular and In vivo Expert in Rare Neurodevelopmental Disorders

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 neurodevelopmental diseases biology, combined with strong in vivo and in vitro skills associated to disease models. The ideal candidate will bring robust hands-on experience in vivo and in vitro skills to support phenotypical assessment of neurodevelopmental disease animal 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 expertise in neurodevelopmental disorders mechanisms and RNA biology

• Design, develop and conduct 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 (fEPSPs), synaptic plasticity measurement techniques, behavior, cognitive and motor functional assays

• In vitro skills: Proficiency in iPSC culture, imaging microscopy, and high throughput imaging microscopy, in vitro neuronal activity (Microelectrodes array plate), Calcium imaging

• Molecular skills: gene expression analysis (qPCR, ddPCR, WB, ELISA/MSD), molecular cloning, vector design, gene editing (e.g., CRISPR)

• Experience with discovery, optimization, and development of small molecules and/or  oligonucleotides