Biomere
Biomere (a JOINN company) is a preclinical CRO based in Worcester, MA. Services include non-GLP PK/PD, discovery toxicology, surgical services, disease models, and laboratory sciences. Surgical services include…
About Oligonucleotide Therapeutics
Antisense oligonucleotides (ASOs) and siRNAs act on RNA, so their pharmacology depends on sequence. A human-specific sequence may not be active in rodents, or even in monkeys, and programs often rely on a nonhuman primate with a matching sequence, or a species-specific surrogate, to show pharmacology and support toxicology species selection.
Oligonucleotides share class-related findings that an experienced CRO will know how to monitor: accumulation in kidney and liver, injection-site reactions, pro-inflammatory effects and, for phosphorothioate ASOs in monkeys, platelet decreases and complement activation. GalNAc-conjugated siRNAs are targeted to the liver, while CNS programs are usually dosed intrathecally or into the cisterna magna because oligonucleotides do not cross the blood–brain barrier.
Bioanalysis is specialized. Hybridization ELISA, LC-MS/MS and PCR-based methods are all used to measure parent oligonucleotide and metabolites in plasma, CSF and tissue, and long tissue half-lives mean study designs often need extended recovery periods.
Oligonucleotide CROs for ASO and siRNA Programs
Showing 1 CRO. Order rotates daily so no lab is permanently listed first.
Questions to ask when choosing an Oligonucleotide Therapeutics CRO
- Which oligonucleotide programs have you supported, ASO, siRNA or both, and through which IND-enabling studies?
- How do you monitor platelets and complement activation in nonhuman primates?
- For CNS programs, do you dose by lumbar bolus or implanted catheter, and how is CSF sampled?
- Which bioanalytical method do you use for our chemistry, and does it measure metabolites as well as parent?
- How do you size recovery groups given long tissue half-lives?