UCB

Splice Bio is a gene therapy company based on technology developed in the Muir Lab at Princeton University. The company is developing novel gene therapies based on its proprietary intein platform technology to treat patients that suffer from incurable genetic diseases. The company’s platform has the potential to address two existing limitations of adeno-associated viruses (AAVs), both by increasing the size of the cargo gene that can be delivered and by expanding the range of tissues that can be targeted.

Neurona Therapeutics Inc. develops cell-based therapies for the treatment of neurological disorders. The company develop therapeutic compositions of specific nerve cells for targeted delivery into the injured nervous system. The company was incorporated in 2008 and is based in South San Francisco, California.

Rinri Therapeutics is a provider of private biotechnology services intended to develop advanced regenerative stem cell-based therapies for the treatment of hearing loss. The company's stem cell technology replaces damaged or dead sensory cells of the inner ear, enabling doctors to restore hearing of patients.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, specializing in the development of adeno-associated viral (AAV) vector technologies and therapeutics aimed at treating rare diseases. Founded in 2015, the company employs a unique STRucture Inspired DEsign approach that merges structural knowledge with accelerated evolution to create innovative AAV capsids. This technology is designed to evade neutralizing antibodies, thereby enhancing gene transfer efficiency. StrideBio's platform supports various gene therapy modalities, including gene addition, gene silencing, and gene editing, with applications across a range of rare genetic disorders.

Nile is an independent company that seeks to improve care for people living with epilepsy, their caregivers, and healthcare providers. The company is on a mission to make the journey of every epilepsy patient predictable. Its digital epilepsy care management platform will provide tools for people living with epilepsy and providers to progress to data-driven, personalized care. Nile is a collaboration-driven organization and is welcoming partners in this mission.

Locanabio, Inc., is an RNA-targeting gene therapy company focused on developing therapies for patients with severe neurodegenerative, neuromuscular, and retinal diseases.

Handl Therapeutics develops and deploys in vivo gene therapy to treat complex neurodegenerative diseases. Handl Therapeutics was incorporated in 2019 and is headquartered in Leuven, Belgium.

Walden Biosciences, Inc, a biotechnology company, develops medicines for individuals living with kidney disease. It targets suPAR, an inflammatory biomarker that connects the kidney to the innate immune system; and Dynamin, a complex enzyme that rebuilds podocytes and the base structure of the kidney. The company was incorporated in 2013 and is based in Cambridge, Massachusetts.

Syndesi Therapeutics is developing molecules that uniquely modulate the synaptic vesicle protein SV2A, which plays a central role in synaptic transmission (the communication between neurons in the brain). Regulating synaptic transmission represents a promising approach to treating Alzheimer’s Disease and other disorders with cognitive impairment. Syndesi has an exclusive license to its platform technology from UCB, the leading company in SV2A research.

ExeVir Bio BV is a Belgium-based biotechnology company established in 2020, focused on developing single-domain antibody therapies to combat viral infections. The company utilizes a llama-derived antibody technology platform, specifically the VHH technology, to create robust antiviral treatments that offer broad protection against coronaviruses, including SARS-CoV-2. ExeVir's lead candidate, VHH72-FC, targets a conserved region on the virus's spike protein essential for cellular entry. This innovative approach not only aims to provide effective treatment and prevention strategies for viral infections but also enables rapid development of drug candidates in response to emerging health threats, ensuring stability and cost-effectiveness for global access. ExeVir Bio's therapies are designed to reduce viral load and inflammation, thereby supporting immune responses and protecting vulnerable populations, including healthcare workers and individuals with co-morbidities.

Engage Therapeutics is a biopharmaceutical company based in Summit, New Jersey, focused on developing innovative rescue therapies for individuals experiencing uncontrolled epileptic seizures. The company's lead investigational product, Staccato® alprazolam, is a handheld drug-device combination that utilizes an FDA-approved delivery system along with the approved medication alprazolam. This product aims to provide a rapid response to active and acute seizures. Preliminary results from a Phase 2a proof of concept study indicated that Staccato alprazolam effectively reduced seizures in a photosensitivity model. Engage Therapeutics plans to advance this product through clinical development using the 505(b)(2) regulatory pathway. The company operates as a subsidiary of UCB SA.

Ra Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics for diseases linked to the complement system's excessive activation. Utilizing a proprietary peptide chemistry platform, the company creates synthetic macrocyclic peptides that possess the specificity of antibodies and the advantageous properties of small molecules. Its primary product candidate, Zilucoplan, is an injectable treatment that has completed Phase II clinical trials for generalized myasthenia gravis and paroxysmal nocturnal hemoglobinuria, as well as a Phase Ib trial for patients with renal impairment. Ra Pharmaceuticals is also exploring pre-clinical programs targeting various complement factors for conditions such as C3 glomerulonephritis and autoimmune diseases. Additionally, the company has a collaboration with Merck & Co. to identify orally available cyclic peptides for cardiovascular targets. Founded in 2008, Ra Pharmaceuticals operates as a subsidiary of UCB SA.

MyHealthTeams is a San Francisco-based company building deeply engaging social networks for people living chronic conditions. MyHealthTeams is leading the way in patient engagement, making it easy for consumers to find and connect with others who understand what they face daily because they have been in their shoes. Eric Peacock & Mary Ray co-founded MyHealthTeams in 2012. As of 2019, MyHealthTeams has built and operates over 30 social networks to serve 90% of the addressable chronic condition population in 13 countries.

Locanabio, Inc., is an RNA-targeting gene therapy company focused on developing therapies for patients with severe neurodegenerative, neuromuscular, and retinal diseases.

Rinri Therapeutics is a provider of private biotechnology services intended to develop advanced regenerative stem cell-based therapies for the treatment of hearing loss. The company's stem cell technology replaces damaged or dead sensory cells of the inner ear, enabling doctors to restore hearing of patients.

Aetion, Inc. develops a science and analytics platform to assess real-world evidence (RWE) from a range of sources. The company offers Aetion Evidence Platform, a platform that generates RWE needed by healthcare decision makers to engage in value-based care. Its solution allows payers, providers, medical device makers, biopharma customers, and academic institutions to analyze data from various clinical and financial interactions in healthcare, including claims, electronic health records, registries, and clinical trials. The company’s platform is also used in database analytics and comparative effectiveness courses taken by epidemiologists, outcomes researchers, health economists, and biostatisticians. It serves customers worldwide. Aetion, Inc. has a strategic collaboration with McKesson. The company was founded in 2012 and is based in New York, New York.

CeriBell, Inc. designs and manufactures medical devices for acquisition and interpreting electroencephalography (EEG) data in patients with various neurological conditions. It offers Ceribell EEG system that includes Ceribell EEG Headband that allows healthcare providers to accommodate various hair types and head sizes; and Ceribell EEG Recorder, a pocket-sized, battery operated recorder that provides clinical quality EEG and on-device EEG display. It serves medical staff in hospital ICUs, inpatient units, and emergency departments. CeriBell, Inc. was formerly known as Brain Stethoscope, Inc. The company was incorporated in 2014 and is based in Mountain View, California.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, specializing in the development of adeno-associated viral (AAV) vector technologies and therapeutics aimed at treating rare diseases. Founded in 2015, the company employs a unique STRucture Inspired DEsign approach that merges structural knowledge with accelerated evolution to create innovative AAV capsids. This technology is designed to evade neutralizing antibodies, thereby enhancing gene transfer efficiency. StrideBio's platform supports various gene therapy modalities, including gene addition, gene silencing, and gene editing, with applications across a range of rare genetic disorders.

Element Genomics is a discovery stage biotechnology company, focused on finding breakthrough cures for severe human disease through the use of functional genomics and manipulation of the epigenome. The company was incorporated in 2015 and is based in Durham, North Carolina.

Beryllium Discovery, LLC is a contract research organization (CRO) based in Bedford, Massachusetts, specializing in drug discovery within the healthcare sector. Founded in 2009, the company collaborates with prominent pharmaceutical firms, innovative biotechnology companies, and academic institutions, leveraging its advanced drug discovery platform to provide comprehensive research services. As a subsidiary of UCB Pharma Ltd since 2017, Beryllium Discovery aims to facilitate the development of novel therapeutics through its collaborative efforts and innovative methodologies.

Clementia Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Montreal, Canada, focused on developing innovative treatments for patients with ultra-rare bone disorders and other diseases characterized by high unmet medical needs. Founded in 2010, the company specializes in disease-modifying therapies, with its lead product candidate being palovarotene, an oral small molecule. Palovarotene has demonstrated significant potential in preventing abnormal bone formation and fibrosis across various tissues. The candidate is currently undergoing evaluation in the Phase III MOVE trial, targeting conditions such as fibrodysplasia ossificans progressive and multiple osteochondromas, as well as dry eye disease. Clementia's commitment to addressing debilitating health issues highlights its role in advancing treatment options for affected patients.

Dermira is a specialty biopharmaceutical company focused on bringing innovative and differentiated products to dermatologists and their patients. Dermira’s portfolio of five product candidates targets significant market opportunities. It was founded in 2010 and headquartered in Menlo Park, California.

Lectus Therapeutics Limited is a UK-based pharmaceutical company. Its mission is to build a development pipeline containing first-in-class ion channel therapeutics identified through the use of its proprietary proteomics technology for pain management and the treatment of urinary bladder disorders.

Synosia Therapeutics develops and intends to commercialise products for unmet medical needs in psychiatry and neurology. The privately-owned company has in its pipeline six clinical-stage compounds acquired through partnerships with Novartis, Roche and Syngenta. Two of the compounds are marketed drugs being tested in new indications to extend their reach into neurological and psychiatric diseases with high unmet medical need, including anxiety and Parkinson's disease.

Schwarz Pharma brings three exciting, approved and late stage new compounds, complementary therapeutic and geographic focus, strong talent and cultural fit.