Sarepta Therapeutics · 19 hours ago
Machine Learning Scientist, Oligo Research Intern
Sarepta Therapeutics is at the forefront of genetic medicine, focusing on Duchenne muscular dystrophy and expanding its capabilities in antisense oligonucleotide therapeutics. The Machine Learning Scientist, Oligo Research Intern will develop machine learning models and a computational framework to enhance ASO design and optimization efforts.
BiotechnologyGeneticsHealth CareTherapeutics
Responsibilities
Develop and implement sequence‑aware machine learning models to prioritize ASO designs by predicted exon‑skipping response across multiple targets
Build a reproducible computational framework including data ingestion, feature engineering, model training, validation, and deployment for oligonucleotide design
Extend modeling and evaluation framework to PMO-gapmers, siRNA PMO hybrids, and conjugate designs to broaden cross‑modality capabilities
Curate and harmonize internal and external literature curated datasets and define robust sequence and structure features such as thermodynamics, accessibility, sequence motifs, secondary structure, and additional context that drive model performance
Establish benchmarks and prospective tests to assess accuracy, robustness, and scalability, and partner with experimental teams to validate predictions
Evaluate and adopt proprietary and open source tools to enhance modeling workflows and accelerate decision support
Maintain a clean and well‑documented codebase, and user guidance for cross‑functional teams
Perform additional related tasks as assigned
Qualification
Required
Current undergraduate or master's student in Computational Chemistry/Biology, Machine Learning, Biomedical/Chemical Engineering, or a related field
Experience in developing and/or adopting probabilistic learning or deep learning models, including Recurrent Neural Networks (RNNs), Graph Neural Networks (GNNs), Transformers, Natural Language Processing models, and Generative AI
Programming and scripting skills in languages such as Python, R, and SQL, with hands-on experience using modern deep learning frameworks such as PyTorch, Tensorflow, skikit-learn or JAX
Experience in developing machine learning models for DNA, RNA, and proteins, including language models, structure prediction, and design
Familiar working with large-scale computing and cloud infrastructures, database systems, and development tools in a production environment
Familiar with data development, tools, and infrastructure: AWS, database technologies, GitHub, GitLab, and Docker containers
Ability to effectively communicate and collaborate with a multidisciplinary team, including chemists, biologists, and data scientists to successfully complete scientific projects
Strong team player with a commitment to continuous learning
Preferred
A background in oligonucleotide design and characterization is strongly preferred
Benefits
Competitive compensation and benefit package
Company
Sarepta Therapeutics
Sarepta Therapeutics focuses on the discovery and development of precision genetic medicine to treat rare diseases.
Funding
Current Stage
Public CompanyTotal Funding
$2.88BKey Investors
Michael Andrew ChambersPharmakon AdvisorsMidCap Financial
2025-12-11Post Ipo Debt· $291.4M
2025-08-21Post Ipo Equity
2025-08-21Post Ipo Debt· $602M
Leadership Team
Recent News
2026-01-24
Genetic Engineering News
2026-01-23
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