A concentrated, high-conviction portfolio
21 positions, updated 30 September 2025
About the company
Agios Pharmaceuticals is a biotechnology company founded in 2008 and headquartered in Cambridge, Massachusetts (US), focused on developing new drugs for genetically defined diseases. Today, the company´s focus is on hemolytic anemias. In 2022 Pyrukynd (Mitapivat) a first-in-class PK activator was approved in the US and EU for the treatment of adults patients with pyruvate kinase (PK) deficiency. The company is currently exploiting α- and β-thalassemia and Sickle Cell Disease as label extension opportunities for the same asset.
About the company
Akero Therapeutics is a biotechnology company founded in 2017 and headquartered in San Francisco, California (US), focused on developing treatments for patients with serious metabolic diseases with a high unmet medical need. The company began with an engineered protein called efruxifermin which they have licensed from Amgen. Efruxifermin is in phase III development for the treatment of MASH which is a serious liver disease caused by metabolic dysregulation. Efruxifermin is being evaluated in a phase III program consisting of three clinical trials.
About the company
Alnylam is a biotechnology company focused on developing RNA therapies, a treatment approach to selectively block the synthesis of specific disease-causing proteins. The company is focusing on four therapeutic areas including genetic medicines, cardio-metabolic diseases, infectious diseases, central nervous system diseases and ocular diseases. Alnylam company was founded in 2002 and headquartered in Cambridge, Massachusetts, in the US. Alongside Ionis, another of BB Biotech´s portfolio companies, Alnylam is considered one of the leaders in the RNA interference field. The company has four products approved and on the market: Patisiran, Givosiran, Lumasiran and Vutrisiran.
About the company
Annexon Biosciences is a biotechnology company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye. Annexon’s approach targets upstream C1q to block the classical complement inflammatory cascade before it starts, and its therapeutic candidates are designed to provide meaningful benefits across multiple autoimmune, neurodegenerative and ophthalmic diseases. Annexon is advancing its mid-to late-stage clinical trials to bring new potential treatments to patients as quickly as possible.
About the company
Argenx is a biotechnology company focused on discovery and development of targeted antibodies through its multiple antibody technology platform. The company is headquartered in Belgium with operations in US and Japan. The company´s lead product Vyvgart (also known as Efgartimod) is marketed for treating generalized Myasthenia gravis. Efgartigimod has the potential to address multiple autoimmune diseases driven by pathological immunoglobulin G. Argenx has several other product candidates in clinical development and can be considered an antibody platform company targeting novel scientific pathways in disease areas with high unmet medical needs.
About the company
Avidity Biosciences is a biotechnology company that focuses on developing RNA-based therapeutics for rare neuromuscular diseases. The company’s work centers around its proprietary AOC™ (Antibody Oligonucleotide Conjugates) platform, which enables targeted delivery of oligonucleotides to specific tissues. Its lead clinical candidate, del-desiran (AOC 1001), is currently in development for the treatment of myotonic dystrophy type 1 (DM1). The pipeline also includes programs for other rare muscle disorders, such as FSHD (facioscapulohumeral muscular dystrophy) with del-brax (AOC 1020), and Duchenne muscular dystrophy (DMD) with del-zota (AOC 1044), particularly for patients amenable to exon 44 skipping. In addition, Avidity is expanding its AOC platform to address other genetic and inflammatory diseases.
About the company
Beam Therapeutics is a biotechnology company focused on developing precision genetic medicines. The company was founded in 2017 and is headquartered in Massachusetts in the US. Beam is working on a new approach to genome engineering termed base editing. Base editors have two principal components that are fused together to form a single protein: (i) a CRISPR protein, bound to a guide RNA, that leverages the established DNA-targeting ability of CRISPR, but modified such that they do not cause a double-stranded break, and (ii) a base editing enzyme, such as deaminase, which carries out the desired chemical modification of the target DNA base. This combination enables the precise targeting and editing of a single base pair of DNA. Beam is building a portfolio of product candidates in disease areas such as hematology, oncology, liver diseases and CNS diseases. Most advanced assets are currently in Phase I/II clinical development for Sickle Cell Disease (BEAM-101) and Alpha-1-Antitrypsin Deficiency (BEAM-302).
About the company
Biohaven is a biotechnology company focused on developing new drugs for a broad range of diseases with a focus on immunology and CNS. The company was founded in 2013 but was relaunched in 2022 following Pfizer´s acquisition of the GCRP portfolio for USD 11.6 billion. Biohaven is headquartered in Connecticut in the US. Biohaven has a pipeline of products in preclinical and clinical development for a broad range of disease areas. The company is developing an extracellular protein degradation platform aiming at leveraging the liver to degrade pathogenic extracellular proteins with implications across multiple autoimmune and cardiac diseases. Furthermore the company is exploring a novel mode of action to treat focal epilepsy by activating a specific potassium channel (Kv7). BHV-7000 is currently starting Phase 2/3 clinical development.
About the company
Celldex Therapeutics is a clinical stage biotechnology company dedicated to developing monoclonal and bispecific antibodies that address devastating diseases for which available treatments are inadequate. The pipeline includes antibody-based therapeutics that have the ability to engage the human immune system and/or directly affect critical pathways to improve the lives of patients with inflammatory diseases and many forms of cancer. The company's lead product candidate Barzolvolimab is a first in class Fc silenced human IgG1 monoclonal antibody (mAb) against wildtype c-KIT receptor leading to depletion and/or inhibition of mast cell activation. The company is on track to start Phase III barzolvolimab clinical development in Chronic spontaneous Urticaria during 2024 and to report Phase II clinical data in Chronic Inducible Urticaria as well as Eosinophilic Esophagitis.
About the company
Edgewise Therapeutics is a biotechnology company founded in 2017 and headquartered in Colorado in the US focused on developing new drugs for muscle diseases. The company is focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is a significant unmet medical need. Edgewise are developing their drug candidates using small molecules that target myosin to treat rare muscle disorders. The company´s technology measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue. Today, the company´s focus is on Duchenne muscular dystrophy, Becker muscular dystrophy and hypertrophic cardiomyopathy (HCM). The company´s lead candidate is EDG-5506 (Sevasemten) is in phase III development for Becker muscular dystrophy and EDG-7500 is in Phase II development for HCM.
About the company
Immunocore is a biotechnology company focused on developing T cell receptor treatments for unmet medical needs in cancer, infectious diseases, and autoimmune diseases. The company was founded in 2008 and is headquartered in Oxfordshire in the UK. Immunocore are enhancing human T cell receptors to overcome the limitations of the natural immune system and current therapeutics approaches, through engineering novel bispecific drugs called ImmTAX molecules. In early 2022 their product Kimmtrak was approved for the treatment of metastatic uveal melanoma. The company has several product candidates in clinical development including IMC-F106C, which is being tested in multiple types of cancer.
About the company
Incyte is a biotechnology company focused on the discovery and development of small-molecule drugs. Founded in 2002, the company is headquartered in the US with operations in Europe and Japan. The company´s leading drug, Jakafi, is marketed and used for the treatment of three indications (myelofibrosis, polycythemia vera, chronic graft-versus-host-disease). Incyte is marketing Jakafi in the US and its partner, Novartis, is marketing the product under the brand name Jakavi elsewhere in the world. Incyte´s other marketed drugs include Opzelura for Vitiligo, Olumiant for rheumatoid arthritis (partnered with Eli Lilly), Iclusig for chronic myeloid leukemia, Pemazyre for cholangiocarcinoma, Tabrecta for lung cancer, and Monjuvi for diffuse large B-cell lymphoma. Incyte´s pipeline includes a broad range of drug candidates within oncology, dermatology, and other disease areas.
About the company
Ionis Pharmaceuticals is a biotechnology company focused on developing RNA-targeted drugs. Its technology platform in the antisense space allows for the control of protein production and reduction at a genetic level. Ionis´ antisense drugs are designed to interact precisely with RNA, allowing them to create treatments that disrupt the disease process and change its course. The company – on its own and in collaboration with its partners – has brought five drugs to the market already and currently has more than ten drug candidates in mid- to late-stage clinical development. The five approved drugs are Spinraza (partnered with Biogen) for spinal muscular atrophy, Tegsedi for hereditary transthyretin amyloidosis polyneuropathy, Waylivra for familial chylomicronemia syndrome, Qalsody (partnered with Biogen) for ALS and Wainua (partnered with AstraZeneca) for polyneuropathy.
About the company
Macrogenics is a biotechnology company focused on the discovery and development of innovative antibody-based therapeutics designed to modulate the immune response for the treatment of cancer. The company was founded in 2000 and is headquartered in Maryland in the US. Macrogenics´ expertise is in the field of protein engineering and the company has developed DART bispecific and Fc Optimization technology platforms. These technology platforms are focused on the creation of antibodies, antibody derivatives, and antibody-like molecules. Macrogenics has multiple product candidates in clinical development with Vobramitamab Duocarmazine, an Antibody Drug Conjugate (ADC) targeting B7H3 for patients with mCRPC being the most advanced one. The currently ongoing Phase II is expected to read-out in 2024.
About the company
Neurocrine Biosciences is a biotechnology company specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. The company lead product Ingrezza (VMAT2 inhibitor) is approved in the US for the treatment of tardive dyskinesia (a condition where patients have involuntary movements that cannot be controlled) and as of August 2023 also for the treatment of Chorea-Huntington’s Disease. On the pipeline side, the company is preparing to file an NDA for crinecerfont, a CRF-R1 antagonist, for the treatment of Congenital Adrenal Hyperplasia (CAH).
About the company
Relay Therapeutics is a clinical-stage precision medicines company transforming the drug discovery process with the goal of bringing life-changing therapies to patients. Built on unparalleled insights into protein motion and how this dynamic behavior relates to protein function, Relay Therapeutics aims to effectively drug protein targets that have previously been intractable, with an initial focus on enhancing small molecule therapeutic discovery in targeted oncology. The company’s Dynamo platform integrates an array of leading-edge experimental and computational approaches to provide a differentiated understanding of protein structure and motion to drug these targets. The company has deployed its Dynamo platform to initially focus on the area of precision oncology. Most advanced clinical candidate is RLY-2608, a pan PI3Kα for the treatment of metastatic breast cancer with a PI3Kα mutation currently in Phase I/II dose expansion in combination with Standard of care.
About the company
Revolution Medicines is a clinical-stage precision oncology company focused on developing novel targeted therapies to inhibit elusive frontier targets within notorious growth and survival pathways, with particular emphasis on RAS and associated signaling pathways. The company is utilizing its deep understanding of genetic drivers and adaptive resistance mechanisms in cancer, coupled with robust drug discovery and medicinal chemistry capabilities, to build a portfolio of compounds that inhibit critical signaling nodes within the RAS and associated pathways. The company is developing a portfolio of mutant-selective RAS inhibitors that could be the first potent, selective, cell-active inhibitors of the active, GTP-bound form of RAS, or RAS(ON). Lead asset is RMC-6236, a multi-selctive KRAS inhibitor that hass shown encouraging signs of antitumor efficacy in lung as well as pancreatic cancer with any type of KRAS mutation.
About the company
Rivus Pharmaceuticals is a non-listed biotechnology company focused on developing a new class of medicines designed to address obesity. The company was founded in 2019 and is headquartered in Charlottesville, Virginia in the US. Rivus is advancing a new class of therapies called Controlled Metabolic Accelerators (CMAs) designed to selectively reduce excess fat by leveraging mitochondrial uncoupling. For its lead program HU6, Rivus has completed Phase I studies and a Phase IIa metabolic study. Rivus recently initiated a Phase IIa study in obese patients with heart failure with preserved ejection fraction and plans to initiate a Phase IIb study in obesity, including a subset of patients with type 2 diabetes, in 2023. Rivus is expanding the therapeutic utility of mitochondrial uncoupling with new CMAs targeting a myriad of diseases where preclinical evidence for safe metabolic acceleration is strong and the therapeutic need is substantial.
About the company
Scholar Rock is a biotechnology company focused on developing monoclonal antibodies that are able to modulate growth factors with extraordinary selectivity. The company was founded in 2012 and is headquartered in Cambridge, Massachusetts, in the US. Scholar Rock has a technology platform based on the understanding of the extracellular activation of growth factors. The company develops antibodies to target the latent, inactive forms of growth factors to selectively and locally modulate growth factor activity within the tissue microenvironment. The lead asset, apitegromab, is an antibody targeting TGF-β currently in Phase III clinical development for Spinal Muscular Dystrophy Type 2 and 3 with data expected in late 2024.
About the company
Vertex is a biotechnology company focused primarily on treatments for the genetic disease cystic fibrosis, as well as other unmet medical needs. Founded in 1989, the company is headquartered in Cambridge, Massachusetts, in the US, with operations across Europe, Australia, and Latin America. The company has a number of approved medicines that treat the underlying cause of cystic fibrosis – a rare and life-threatening genetic disease. The approved drugs in this disease area include Kalydeco, Orkambi, Symdeko, and Trikafta. Besides cystic fibrosis, Vertex has a pipeline of investigational small molecule medicines for other serious diseases where it has deep insight into causal human biology, including alpha-1 antitrypsin deficiency, APOL1-mediated kidney diseases, and pain. In addition, the company has a partnership with Crispr Therapeutics for the development and commercialization of Casgevy, the first ex-vivo cell therapy ever been approved by the US FDA (December 2023) for patients with sickle cell therapy.
About the company
Wave Life Sciences is a clinical-stage company focused on developing oligonucleotide-based therapeutics for devastating rare diseases. Wave Life Sciences’ proprietary discovery and drug development platform, PRISM, enables the precise design, optimization, and production of novel stereo-pure oligonucleotides across multiple therapeutic modalities, including RNA editing, splicing, and silencing. WVE’s current drug candidates include WVE-006 (RNA editing for alpha-1-antitrypsin deficiency), WVE-N531 (exon 53 skipper for Duchenne muscular dystrophy), WVE-003 (antisense oligonucleotide selective for mutant huntingtin in Huntington’s disease) and a new candidate targeting INHBE to treat metabolic disorders including obesity.
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