CDMOs supporting Rare Disease programs
Rare Disease is an important therapeutic indication used by sponsors to shortlist partners for specific delivery, regulatory, and execution needs. This page groups CDMO organizations supporting programs in Rare Disease, so you can quickly identify which teams align with your program priorities without having to scan unrelated profiles.
Use this listing as a starting point: open each profile to review scope, facility footprint, and public evidence that supports the listed capabilities. Because the directory is fit-first, the goal is not to crown one universal "best" provider, but to help you build a right-sized shortlist for your stage, geography, and quality expectations.
When you are ready to compare options, move to search and add filters for adjacent criteria such as modalities, indications, and claim status. Combining this indication area view with those filters makes it easier to narrow quickly from discovery to outreach while keeping the process transparent and reproducible.
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Matching companies
- Aldevron
Aldevron is a CDMO specializing in mRNA manufacturing with LNP encapsulation and fill/finish, viral vector production, plasmid DNA, and gene therapy solutions from discovery through commercialization.
- Andelyn Biosciences
CDMO specializing in plasmid DNA and AAV viral vector manufacturing for cell and gene therapy, with digital-first platform emphasizing data integrity and collaboration.
- AskBio
AskBio is a clinical-stage gene therapy company developing AAV gene therapies for genetic and rare disorders, with a focus on advancing genetic technology and lowering production costs to increase patient access.
- Cell and Gene Therapy Catapult
UK-based innovation and manufacturing support organization for cell and gene therapies, offering infrastructure, expertise, and collaboration across the ATMP lifecycle.
- ElevateBio
Advanced therapies CDMO providing discovery, process development, manufacturing, and regulatory support for cell therapies, viral vectors, mRNA, and gene editing programs from preclinical through commercial scale.
- Forge Biologics
Dedicated AAV gene therapy CDMO with a 200,000+ sq. ft. cGMP facility featuring 20 suites and bioreactors up to 5,000L, supporting 60+ clients across 35+ indications with high-yield manufacturing and seamless regulatory compliance.
- LGC Biosearch Technologies
GMP-compliant manufacturer of custom oligos, qPCR probes, and diagnostic kits for infectious diseases, oncology, and prenatal testing; ISO 13485-certified for clinical and diagnostic markets.
- Maravai LifeSciences
Leading provider of nucleic acid products, custom enzymes, and bioprocess analytics supporting vaccine, therapeutic, and diagnostic development from discovery through commercialization, with mRNA CDMO services including proprietary CleanCap® capping technology.
- Pharmaron
Premier life science CRO and CDMO offering integrated R&D and manufacturing across drug discovery, preclinical, clinical development, and commercialization for small molecules, biologics, and cell & gene therapies. 25,000+ employees across China, US, and UK.
- Syngene International
Innovation-led CRDMO offering integrated scientific services from early discovery through commercial manufacturing, with cGMP-compliant facilities, 5,200+ scientists, and a new 50,000-liter single-use bioreactor capacity in the U.S.
- Viralgen
Viralgen is a CDMO specializing exclusively in cGMP AAV gene therapy manufacturing, with the world's largest dedicated AAV production capacity and a 100% success rate for IND/IMPD dossiers across 50+ gene therapy customers.
Markdown mirror: /indications/ind_rare_disease.md