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.
Personalised cancer treatment selection using in vivo drug screening
Vivan Therapeutics is a London-based biotech developing a personalised cancer therapeutics platform. The company's Personal Discovery Process sequences a patient's tumour genome,…
Bicistronic AAV gene therapies for retinal disease
Ikarovec is a near-clinical-stage gene therapy company developing bicistronic AAV-based therapeutics for vision-threatening retinal diseases. Its proprietary platform combines two…
AAV gene therapy targeting the seizure focus in refractory epilepsy
EpilepsyGTx is a biotechnology company developing gene therapies for focal refractory epilepsy, a condition in which seizures arise from a specific brain region and do not respond…
Gene therapies targeting the ageing process and age-related disease
Genflow Biosciences is a longevity-focused biotechnology company developing gene therapies designed to slow or halt the biological ageing process and delay age-related disease.…
Clinical genomic decision support software for rare disease and oncology
Congenica is a clinical genomic interpretation platform spun out of the Wellcome Sanger Institute. Its software supports variant analysis and clinical reporting across rare…
ADC oncology company developing novel spliceosome-modulating payloads
Akari Therapeutics is a clinical-stage biopharma company developing next-generation bi-functional antibody-drug conjugates for cancer treatment. Its proprietary ADC platform…
CAR-T and bispecific antibody therapies for blood cancers
Hemogenyx Pharmaceuticals is a clinical-stage biopharmaceutical company developing targeted therapies for haematological malignancies and autoimmune diseases. Its lead asset,…
siRNA gene therapies targeting root causes of ALS and frontotemporal dementia
Crucible Therapeutics Limited is a preclinical-stage biotechnology company developing siRNA-based gene therapies for Amyotrophic Lateral Sclerosis and Frontotemporal Dementia…
Global biopharma with UK R&D centre on the Wirral Peninsula
Bristol-Myers Squibb (BMS) is a global biopharmaceutical company with a long-established UK R&D site at Moreton on the Wirral Peninsula and its UK and Ireland commercial…
Clinical-stage rare disease biotech acquiring deprioritised pharma assets
Mereo BioPharma is a clinical-stage biopharmaceutical company focused on developing treatments for rare diseases. Founded in 2015, its business model centres on acquiring…
Antisense oligonucleotide gene therapies for rare childhood leukodystrophies
SynaptixBio is a rare disease biotech company developing gene therapies for severe leukodystrophies and childhood neurodegenerative diseases, using antisense oligonucleotide…
siRNA therapeutics targeting liver-expressed disease genes
Silence Therapeutics is a clinical-stage biotechnology company developing siRNA therapeutics using its proprietary platform. The platform enables precise silencing of…