Wave seeks Scientists-I for Cambridge, MA location. Duties include:

Research Leadership & Competitive Intelligence

• Conduct competitive intelligence research to evaluate targets for novel RNA therapies. Analyze scientific literature, industry reports, and competitive landscapes to identify novel disease targets and assess feasibility for antisense oligonucleotide (ASO) intervention. (10%)
• Lead target discovery and validation programs by designing and overseeing experiments that investigate target biology, mechanism of action, and therapeutic potential of ASO candidates. Work closely with cross-functional teams to prioritize and advance promising targets. (10%)

Cross-functional & Strategic Leadership

• Lead and manage multifunctional teams by driving collaboration between Chemistry, Biology, DMPK, and Toxicology teams to develop comprehensive preclinical strategies to advance ASO candidates. Serve as the scientific lead for target programs. (10%)
• Participate in strategic decision-making with senior leadership on ASO program viability, target selection, and competitive positioning. Present findings to executive stakeholders to shape research priorities. (5%)

Preclinical & Translational Research

• Independently lead and direct preclinical research programs to advance ASO therapeutics for rare and severe neurodegenerative diseases including Ataxias and other CNS indications. Define project scope, experimental strategy, and key milestones. (10%)
• Manage and prioritize multiple in vivo pharmacology studies by defining study designs, analyzing PK/PD relationships, and driving strategic decision-making for candidate selection and optimization. Serve as primary point of contact for internal and external study partners. (10%)
• Oversee development of new high-throughput in vitro assays to assess potency, efficacy, and cellular uptake of ASO therapeutics in patient-derived iPSCs, primary neurons, and astrocytes. Establish best practices and improve throughput efficiency. (8%)

Regulatory & External Engagement

• Drive regulatory and scientific documentation, including drafting, reviewing, and finalizing study reports, Standard Operating Procedures (SOPs), and regulatory submissions and ensure compliance with industry standard and regulatory requirements. (10%)
• Represent company at external scientific meetings, conferences, and partner collaborations to communicate study results, scientific insights, and research milestones. (10%)

Mentorship & Innovation

• Mentor junior scientists, research associates, and cross-functional team members by providing technical guidance, performance feedback, and professional development support. (8%)
• Drive new technology development initiatives by identifying and implementing novel methodologies for ASO delivery, target engagement, and efficacy assessment. (4%)

Laboratory Operations
Manage procurement and evaluation of external research tools, reagents, and technologies to optimize lab workflows and support experimental execution. (5%)
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Position requires MS or foreign equivalent in Biotechnology, Molecular Biology, Neuroscience, Pharmacology, or related field and 4 years’ experience in nucleic acid therapeutic research and development of antisense oligonucleotides (ASOs) for central nervous system (CNS) and neurodegenerative disorders. Experience must include the following (may be gained concurrently):

• 4 years’ experience in competitive early stage research and screening of therapeutic targets for RNA and nucleic acid-based medicines to address severe neurodegenerative conditions.
• 4 years’ experience designing and implementing experimental models for therapeutic target evaluation and validation and designing, executing, and analyzing large-scale SAR

Relationship (SAR) screens for ASOs utilizing transcriptomic and proteomic approaches to assess RNA-based interventions, optimizing SAR screening conditions across multiple cellular models, identifying lead compounds, and refining lead progression strategies for CNS and neuromuscular disorders.

• 4 years’ experience developing and performing mechanistic studies to assess oligonucleotide activity including transcript and protein-level evaluations using RNA-seq, qPCR, and hybridization-based assays.
• 4 years’ experience leading design and execution of in vitro PD, PK, and biodistribution studies and assessments in mouse models for ASO therapeutic candidates.
• 4 years’ experience working with iPSCs, NPCs, and primary neurons including differentiation into disease-relevant neuronal subtypes for ASO screening and validation.
• 4 years’ experience performing quantitative protein analysis for oligonucleotide therapeutics including ELISA, Western blotting, and amplicon sequencing to measure ASO-mediated protein modulation in CNS and peripheral tissues, and generating RNA and protein analysis workflows such as BRAVO (liquid handler), qPCR and MSD to assess drug-target interactions.
• 4 years’ experience collaborating with cross-functional teams such as chemistry, toxicology, and DMPK groups to optimize lead candidate selection, analyze biodistribution results, and prepare preclinical data packages.
• 4 years’ experience investigating impact of chemical modifications on target engagement, allele selectivity, potency, and duration utilizing stereopure phosphorothioate (PN) chemistry platform.
• 4 years’ experience utilizing amplicon sequencing pipelines to validate allele selectivity in ASO-treated samples including SNP phasing, detection of allele-specific expression, and validation of knockdown selectivity in preclinical disease models.
• 2 years’ experience leading large-scale preclinical oligo PK/PD studies to determine IC50 values, assess drug distribution, target engagement levels, and clearance in CNS and peripheral tissues, and generate critical dose-response data to support ASO therapies preclinical development .
• 2 years’ industry experience preparing and reviewing regulatory documentation, including SOPs, preclinical study reports, and IND filings.
• 2 years’ experience mentoring junior scientists in drug study design, bioassay development, and data interpretation using statistical tools such as GraphPad and JMP and implementing workflow optimizations such as KNIME workflow and ThinkCell software to enhance experimental reproducibility.
• 1 year experience presenting findings at scientific meetings, and/or external collaborations, including contributions to peer-reviewed publications.
• 6 months experience applying molecular biology techniques including RNA/DNA extraction, PCR and gel electrophoresis for at least 50 experiments in laboratory setting.
• 6 months’ experience performing computational analysis of sequencing data using BLAST, SnapGene and Primer3 for sequence design and data interpretation.

Please refer the Job description for details