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 dedicated to enhancing care for individuals living with epilepsy, along with their caregivers and healthcare providers. The company aims to create a predictable journey for epilepsy patients through its digital care management platform. This platform offers tools that enable patients to manage their medication regimens, receive personalized alerts related to seizures and side effects, and access the latest educational resources on epilepsy. By focusing on data-driven, personalized care, Nile fosters a collaborative environment and actively seeks partnerships to further its mission of improving the quality of life for those affected by epilepsy.

Locanabio, Inc. develops RNA-targeted gene therapies to treat a spectrum of underserved diseases. It offers therapies for neuromuscular, neurodegeneration, and ophthalmology diseases. Locanabio, Inc. was formerly known as Locana, Inc. and changed its name to Locanabio, Inc. in July 2020. The company was founded in 2016 and is based in San Diego, California.

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 SA is a biopharmaceutical company based in Louvain-la-Neuve, Belgium, founded in 2018. The company focuses on developing drug molecules that modulate the synaptic vesicle protein SV2A, which is crucial for synaptic transmission—the process that facilitates communication between neurons in the brain. By regulating this transmission, Syndesi aims to create effective treatments for Alzheimer’s Disease and other cognitive impairment disorders. The company holds an exclusive license to its innovative platform technology from UCB, a leader in SV2A research. Through its efforts, Syndesi Therapeutics seeks to provide healthcare providers with advanced therapeutic options for managing these challenging conditions.

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, based in Summit, New Jersey, is a biopharmaceutical company focused on developing innovative therapies for individuals experiencing uncontrolled epileptic seizures. The company's lead product, Staccato® alprazolam, is a handheld drug-device combination that utilizes an established FDA-approved delivery system to administer alprazolam, an FDA-approved benzodiazepine. This product aims to provide immediate rescue therapy for active seizures, offering a potential solution for patients and their caregivers. In a Phase 2a proof of concept study, Staccato alprazolam demonstrated efficacy in reducing seizures in a photosensitivity model. Engage Therapeutics plans to advance this product into further clinical development through a 505(b)(2) regulatory pathway, positioning it as a promising option for seizure management.

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, Inc. is a San Francisco-based company that creates online social networking platforms for individuals living with chronic health conditions and those who care for them. Founded in 2011, the company operates various platforms, including MyAutismTeam, MyBCTeam, MyMSTeam, and MyCrohnsandColitisTeam, designed to foster connections among users who share similar health challenges. By facilitating these connections, MyHealthTeams enhances patient engagement, allowing users to find support and share experiences with others who understand their daily struggles. The company has developed over 30 distinct social networks, serving approximately 90% of the chronic condition population across 13 countries.

Locanabio, Inc. develops RNA-targeted gene therapies to treat a spectrum of underserved diseases. It offers therapies for neuromuscular, neurodegeneration, and ophthalmology diseases. Locanabio, Inc. was formerly known as Locana, Inc. and changed its name to Locanabio, Inc. in July 2020. The company was founded in 2016 and is based in San Diego, 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.

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. specializes in the design and manufacture of medical devices aimed at acquiring and interpreting electroencephalography (EEG) data for patients with neurological conditions. The company offers the Ceribell EEG System, which includes a versatile EEG headband accommodating various hair types and head sizes, and a compact, battery-operated EEG recorder that delivers clinical-quality data with an on-device display. Ceribell focuses on improving the accessibility and efficiency of EEG technology by developing an FDA-cleared instant EEG system that can be set up by any healthcare provider in just six minutes, eliminating the need for specialized technologists. This system features a unique Brain Stethoscope function that converts brainwaves into sound, allowing for the detection of seizures through auditory means. By facilitating earlier diagnoses and targeted treatments for seizures, including non-convulsive seizures, Ceribell aims to enhance patient outcomes while reducing risks such as mortality and complications. Founded in 2014 and based in Mountain View, California, Ceribell is committed to transforming the diagnosis and management of serious neurological conditions in acute care settings.

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 dedicated to drug discovery and development, specializing in next-generation ion channel modulators. The company aims to create a robust pipeline of first-in-class therapeutics, primarily targeting pain management and urinary bladder disorders. Utilizing proprietary proteomics technology, Lectus Therapeutics focuses on developing innovative treatments for various conditions, including angina, asthma, and epilepsy, alongside its core areas of interest. Through its commitment to advancing ion channel therapeutics, the company seeks to address unmet medical needs in the pharmaceutical landscape.

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.