Violet Research Institute · 2 months ago
Scientific Director, Neuro-therapeutics
United States
Full-time
Remote
Director/Executive
$175K/yr - $250K/yrViolet Research Institute (VRI) is focused on advancing personalized medicine for genetic diseases. They are seeking a Scientific Director for Neuro-therapeutics to lead the development of a new therapeutic program, coordinating a cross-functional team and overseeing scientific strategy, target selection, and regulatory submissions.
Biotechnology
Responsibilities
Lead target identification and sequence prioritization for VRI’s therapeutic program, including known causative genes and emerging targets relevant to the disease biology
Determine therapeutic modality — ASO vs. siRNA, backbone chemistry, modification strategy — with scientific rationale grounded in both biology and practical development considerations
Evaluate sequence candidates, apply scoring frameworks, and converge on lead molecules most likely to succeed in CNS delivery
Define Plan B and Plan C candidate strategies to ensure program progress is never contingent on a single compound
Translate target biology insights into actionable design criteria for the synthesis and screening pipeline
Provide scientific direction for ASO and siRNA design, guiding vendor partners and internal scientists on backbone chemistry, modification strategy, and design tradeoffs
Lead design reviews, evaluate screening data, and make candidate advancement and termination decisions in coordination with the CSO
Assess in vitro and in vivo datasets against advancement criteria, identifying signal from noise and recommending next steps
Stay current on the relevant oligonucleotide literature and competitive landscape, flagging findings that should influence VRI’s strategy
Define the scientific questions that in vitro campaigns must answer, including cell-based assays, disease-relevant neuronal models, and functional readouts appropriate to the target biology
Evaluate in vivo study designs for scientific rigor and IND-relevance; interpret results in context of regulatory requirements
Provide scientific oversight to external labs and CROs on study design and endpoint validity
Oversee manufacturing of ASO and siRNA candidates, ensuring designs meet research-scale synthesis requirements and can be scaled appropriately toward GMP production
Bring CMC awareness to program planning, anticipating manufacturing constraints that could affect IND timelines
Own the scientific content of the IND package: nonclinical study rationale, pharmacology and toxicology summaries, and the scientific narrative that ties target biology to clinical hypothesis
Make or delegate all scientific judgment calls required for IND sections; what data is sufficient, what gaps require additional studies, and what the FDA will scrutinize on the nonclinical side
Ensure all nonclinical studies are designed and executed in alignment with IND-enabling standards and FDA expectations for rare disease therapeutics
Engage with expedited regulatory pathways (Fast Track, Orphan Drug, Expanded Access) as appropriate, and maintain contingency pathway awareness across alternative jurisdictions
Qualification
Required
Advanced degree (PhD or equivalent) in medicinal chemistry, biochemistry, molecular biology, or a related field
Direct involvement in oligonucleotide drug development like ASOs, siRNAs, or closely related modalities. You understand the chemistry (backbone modifications, 2'-MOE, LNA, PS), the biology (RNase H, RISC, off-target risks), and the practical realities of moving these molecules through a development pipeline. You do not need to have been the only person on a program, but you need to have been close enough to the work to have an informed opinion on it
Expertise with oligonucleotide modalities including gapmers, SSOs, siRNA, and saRNA
Demonstrated experience leading or coordinating cross-functional drug development work, including managing external partners toward shared milestones
Preferred
Background from organizations with strong oligonucleotide platforms, particularly those with CNS program experience
Prior experience serving as a scientific lead or Compound Development Team Leader (CDTL) in an integrative program role
Experience working on or managing rare disease programs
Experience working with patient foundations or advocacy organizations where family urgency creates non-negotiable deadlines
Benefits
Full suite of benefits
Company
Violet Research Institute
Building the future of personalized medicine for patients with genetic diseases.
2-10 employees
Funding
Current Stage
Early StageCompany data provided by crunchbase