Senior Scientist Preclinical Research and Discovery

Profile

Reporting to the Associate Director of Preclinical, this position will be responsible for key aspects of the discovery and development of **MEMBERS ONLY**SIGN UP NOW***.’s Enhance Delivery Oligonucleotide (EDO) programs and future pipeline product candidates as well as interfacing with **MEMBERS ONLY**SIGN UP NOW***.’s external CRO partners. Further, the successful candidate must be able to effectively multitask, have strong documentation skills, and should be comfortable working in multidisciplinary, cross-functional teams.

Responsibilities:

• Develop, optimize and perform in vitro efficacy and tolerability assays to assess **MEMBERS ONLY**SIGN UP NOW***.’s enhanced delivery oligonucleotides.
• Work collaboratively with pre-clinical, discovery and CMC teams to drive the development and optimization of **MEMBERS ONLY**SIGN UP NOW***.’s pipeline therapeutics to ensure timely delivery of key datasets.
• Identify CRO partners and subsequently oversee outsourced in vitro and in vivo studies.
• Maintain clear technical records and prepare summary presentations and reports of ongoing activities for both internal and external audiences.
• Foster a culture of collaboration, innovation, discovery, and cutting-edge research culture focused on scientific excellence, open communication, and continual improvement.
• Mentoring junior lab members.

Requirements:

• MS or PhD in life science with at least 2-5 years of relevant experience in the pharmaceutical industry.
• Experience with in vitro disease models using primary and immortalized human cells and medium-through put assay formats to support drug discovery.
• Proficient in techniques, including method development and optimization, critical for molecular and cellular biology experiments e.g. protein and nucleic acid extraction and gene expression analysis by RT-PCR, qPCR and/or digital PCR and western blotting
• Experience with managing and/or conducting studies with CROs.
• Demonstrated experience and expertise in skeletal and/or cardiac muscle biology.
• Experience working with cell penetrating peptides, nucleic acids, and/or oligonucleotide conjugates in drug discovery and development.
• Proven ability to work independently, think strategically and be self-motivated.
• Excellent communication and presentation skills to efficiently relay information to project teams, senior management team and with other key stakeholders.
• Experience working in a start-up or small biotech environment is a plus.

About **MEMBERS ONLY**SIGN UP NOW***.:

We are a biotechnology company advancing next-generation oligonucleotide therapies for neuromuscular and neurological diseases. Our ambition is to unlock the potential of nucleic acid therapeutics by leveraging the drug delivery capabilities of our proprietary Enhanced Delivery Oligonucleotide (EDO) platform. We are applying this technology across a wide range of indications through independent and partnered development pathways and are rapidly growing our team, especially at our U.S. headquarters in Boston, Massachusetts.

**MEMBERS ONLY**SIGN UP NOW***. is proud to be an equal opportunity workplace and is an affirmative action employer.

**MEMBERS ONLY**SIGN UP NOW***. requires all employees to be fully vaccinated for COVID-19 prior to starting work. The CDC defines fully vaccinated as two weeks after the second dose for Pfizer and Moderna, and two weeks after the single dose of Johnson & Johnson. **MEMBERS ONLY**SIGN UP NOW***. will require proof of vaccination and consider requests for exemption from this requirementas a reasonable accommodation for medical reasons or sincerely held religious beliefs where the accommodation would not cause **MEMBERS ONLY**SIGN UP NOW***. undue hardship or pose a direct threat to the healthorsafety ofthe employee orothers.

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