Rare disease companies develop diagnostics, therapies and research technologies for conditions affecting relatively small patient populations. Their work often focuses on inherited disorders, complex diagnostic pathways and diseases with limited or no existing treatment options.
This category includes companies working on:
Browse 128 UK rare disease companies below.
Clinical-stage biopharma targeting cancer immunotherapy and obesity
Poolbeg Pharma is a clinical-stage biopharmaceutical company developing small molecule therapies for high unmet medical needs. Its lead programme, POLB 001, is a p38 MAP kinase…
Engineered therapeutic red blood cells for rare metabolic and other diseases
Scarlet Therapeutics is a biotechnology company developing a platform for generating therapeutic red blood cells (tRBCs). Erythroid cell lines are genetically engineered to…
Small molecule inhibitors targeting somatic expansion in neurodegeneration
LoQus23 Therapeutics is a biotechnology company developing oral small molecule inhibitors of DNA mismatch repair for the treatment of Huntington's disease and other triplet repeat…
Full-service CRO specialising in oncology and early-phase trials
Pivotal CRO is a full-service contract research organisation providing end-to-end clinical development support across all trial phases, from first-in-human to Phase III. The…
TCR bispecific immunotherapies for cancer and infectious disease
Immunocore is a commercial-stage biopharma developing T cell receptor bispecific immunotherapies. Its lead product tebentafusp received FDA approval in 2022 for metastatic uveal…
AstraZeneca's rare disease arm targeting the complement cascade
Alexion, AstraZeneca Rare Disease is the rare disease group within AstraZeneca, formed following the 2021 acquisition of Alexion Pharmaceuticals. With nearly 30 years of rare…
Antibody therapy to deplete amyloid deposits in ATTR cardiomyopathy
Immutrin is a biotechnology company developing an engineered antibody therapeutic designed to actively remove amyloid deposits from tissues in patients with amyloidosis. Its lead…
In vivo cell reprogramming therapies for degenerative disease
Mogrify is a biotechnology company developing a novel class of in vivo reprogramming therapies for degenerative diseases. Its proprietary platforms use large-scale transcriptomic…
Autophagy-enhancing small molecule drugs for neurodegeneration and rare disease
Samsara Therapeutics is a drug discovery company developing first-in-class small molecule therapeutics that restore autophagy, the cellular process of degrading and recycling…
European hub for RNAi therapeutics developer
Alnylam UK is the European operational headquarters of Alnylam Pharmaceuticals, the pioneer of RNA interference therapeutics. Based in Maidenhead, the UK office serves as…
AI platform integrating biology, chemistry and automation for drug discovery
Recursion is a clinical-stage biotechnology company using AI and robotic wet-lab automation to industrialise small molecule drug discovery. Its platform integrates phenomics,…
AI-driven combinatorial analytics platform for chronic disease biology
PrecisionLife is a computational biology company applying AI-driven combinatorial analytics to complex chronic diseases. Its platform identifies causal biological mechanisms and…