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Orphan Therapeutics

Rare Disease Research Platform

Accelerating Drug Discovery for Rare Diseases

Advanced computational platform combining Python, Fortran, and FPGA acceleration for protein analysis, genomic research, and gene therapy development

Platform Capabilities

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Protein Structure Prediction

AlphaFold-style algorithms with Fortran acceleration for rapid protein folding predictions

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Genomic Variant Analysis

Identify pathogenic mutations and analyze disease associations in rare disease genes

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Virtual Drug Screening

Screen thousands of compounds against target proteins with FPGA acceleration

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Molecular Dynamics

High-performance protein simulations using Fortran for 10-100x speedup

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CRISPR/Cas9 Design

Design guide RNAs with efficiency prediction and off-target analysis

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AAV Vector Design

Optimize AAV vectors for tissue-specific gene therapy delivery

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Base & Prime Editing

Precise single nucleotide corrections and versatile gene editing

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Clinical Trials Data

Access information on approved gene therapies and active clinical trials

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FPGA Acceleration

Ultra-fast genomic sequence alignment and drug screening (optional hardware)

Gene Therapy Development Tools

āœ‚ļø CRISPR/Cas9 Tools

  • • Guide RNA design with efficiency prediction
  • • Genome-wide off-target analysis
  • • Multiple Cas variants (SpCas9, SaCas9, Cas12a)
  • • PAM sequence optimization

🦠 AAV Vector Design

  • • Tissue-specific serotype selection
  • • Promoter optimization
  • • Packaging capacity analysis
  • • Clinical-grade production guidelines

šŸŽÆ Base & Prime Editing

  • • CBE/ABE for C→T and A→G conversions
  • • Prime editing for insertions/deletions
  • • Minimal off-target effects
  • • No double-strand breaks required

šŸ„ Clinical Data

  • • 4 FDA/EMA approved therapies
  • • 228+ active clinical trials
  • • Real-world efficacy data
  • • Safety and monitoring information

Performance Benchmarks

TaskPython+ Fortran+ FPGA
MD Simulation (1000 atoms)2h12min3min
Sequence Alignment45min10min45s
Drug Screening (10k compounds)8h1h10min