An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation of the country.
AstraZeneca plc (AZ) is a British-Swedish multinational pharmaceutical and biotechnology company with its headquarters at the Cambridge Biomedical Campus in Cambridge, England. It has a portfolio of products for major diseases in areas including oncology, cardiovascular, gastrointestinal, infection, neuroscience, respiratory, and inflammation. It was involved in developing the Oxford–AstraZeneca COVID-19 vaccine.
The Medical Research Council (MRC) is responsible for co-coordinating and funding medical research in the United Kingdom. It is part of United Kingdom Research and Innovation (UKRI), which came into operation 1 April 2018, and brings together the UK's seven research councils, Innovate UK and Research England. UK Research and Innovation is answerable to, although politically independent from, the Department for Business, Energy and Industrial Strategy.
Sir Gregory Paul Winter is a Nobel Prize-winning English molecular biologist best known for his work on the therapeutic use of monoclonal antibodies. His research career has been based almost entirely at the MRC Laboratory of Molecular Biology and the MRC Centre for Protein Engineering, in Cambridge, England.
MedImmune, LLC was a wholly owned subsidiary of AstraZeneca before February 14, 2019, when it was announced that the MedImmune name and branding would be discontinued in favor of AstraZeneca.
The Cambridge Biomedical Campus is the largest centre of medical research and health science in Europe. The site is located at the southern end of Hills Road in Cambridge, England.
Sir Menelaos (Mene) Nicolas Pangalos is a British neuroscientist of Greek descent.
MorphoSys AG is a German biopharmaceutical company founded in 1992. The company is headquartered near Munich, Germany, and has a wholly owned subsidiary, MorphoSys US Inc., in Boston, Massachusetts, in the US. The company has various antibody, protein and peptide technologies that it uses to discover and develop both proprietary and partnered drug candidates. The company has more than 100 drugs in its wider pipeline that are being investigated for a variety of diseases. While many of these are being developed in partnership with pharma and biotech companies, MorphoSys also has a proprietary pipeline with a focus on cancer and autoimmune diseases.
Cambridge Antibody Technology Group Plc, was a biotechnology company headquartered in Cambridge, England, United Kingdom. Its core focus was on antibody therapeutics, primarily using Phage Display and Ribosome Display technology.
The pharmaceutical industry in the United Kingdom directly employs around 73,000 people and in 2007 contributed £8.4 billion to the UK's GDP and invested a total of £3.9 billion in research and development. In 2007 exports of pharmaceutical products from the UK totalled £14.6 billion, creating a trade surplus in pharmaceutical products of £4.3 billion.
Brodalumab, sold under the brand name Siliq in the US and Kyntheum in the EU, is a human monoclonal antibody designed for the treatment of inflammatory diseases.
Moxetumomab pasudotox, sold under the brand name Lumoxiti, is an anti-CD22 immunotoxin medication for the treatment of adults with relapsed or refractory hairy cell leukemia (HCL) who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. Moxetumomab pasudotox is a CD22-directed cytotoxin and is the first of this type of treatment for adults with HCL. The drug consists of the binding fragment (Fv) of an anti-CD22 antibody fused to a toxin called PE38. This toxin is a 38 kDa fragment of Pseudomonas exotoxin A.
Tralokinumab sold under the brand names Adtralza (EU/UK) and Adbry (US) among others, is a human monoclonal antibody used for the treatment of atopic dermatitis. Tralokinumab targets the cytokine interleukin 13.
CompugenLtd. is a clinical-stage publicly traded predictive drug discovery and development company headquartered in Israel, with shares traded on the NASDAQ Capital Market and on the Tel Aviv Stock Exchange. Compugen was established as computational drug discovery service provider in 1993. Compugen originally acted as service provider for pharma companies, supplying its software and computational services to predict different types of biological phenomena. It had arrangements with big companies such as Novartis AG, Abbot Laboratories and Pfizer Inc. Subsequently, Compugen made a decision to become a drug development company with its own internal pipeline, and in 2010, decided to a focus on oncology and immunology. OncoMed Pharmaceuticals and Five Prime Therapeutics are among Compugen's competitors.
AbbVie Inc. is an American pharmaceutical company headquartered in North Chicago, Illinois. It is ranked sixth on the list of largest biomedical companies by revenue. In 2023, the company's seat in Forbes Global 2000 was 74, and rank 89 on the 2024 list. The company's primary product is Humira (adalimumab), administered via injection. It is approved to treat autoimmune diseases including rheumatoid arthritis, Crohn's disease, plaque psoriasis, and ulcerative colitis.
A royalty fund is a category of private equity fund that specializes in purchasing consistent revenue streams deriving from the payment of royalties. Royalties are a usage-based payment from one individual or entity to another individual or entity, giving the right to use an asset, product, service or idea.
Samsung Biologics Co., Ltd. is a global contract development and manufacturing organization headquartered in Songdo, Incheon, South Korea. The biotech division of Samsung Group, its core services range from late discovery to large-scale commercial manufacturing. The company focuses on monoclonal antibodies, bispecific antibodies, antibody-drug conjugates, and mRNA vaccines.
Optimer ligands are short synthetic oligonucleotide molecules composed of DNA or RNA that bind to a specific target molecule. They are engineered to bind their target molecules with affinity typically in the low nanomolar range. Optimers can be used as antibody mimetics in a range of applications, and have been optimized to increase their stability, reduce their molecular weight, and offer increased scalability and consistency in manufacture compared to standard aptamer molecules.
Jane Osbourn, OBE, is a scientist and former chair of the UK BioIndustry Association.
AION Labs is an Israeli venture studio focused on the adoption of artificial intelligence (AI) and machine learning (ML) in pharmaceutical discovery and development processes.
