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
** 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  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.
*
* 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 (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
* Familiarity with drug discovery and development processes
** Skills and competencies:
*** 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…