List of RNA Therapies Companies in Switzerland - 4

HAYA Therapeutics is a precision medicines company developing RNA-guided programmable therapeutics targeting the regulatory genome for serious health conditions including cardiovascular disease and cancer. The company is using its innovative cell-state modifying platform to gain novel insights into the biology of long non-coding RNAs (lncRNAs), the ‘dark genomes’ information processing features and ‘source code’. HAYA’s lead therapeutic candidate is HTX-001, an antisense oligonucleotide targeting Wisper, which is a tissue and cell-specific cardiac lncRNA known to play a role in heart failure. The company is also developing a pipeline of lncRNA-targeting candidates for the cell-specific treatment of diseases in other tissues, including the lungs and the microenvironment of solid tumor cancers. Headquartered at the life sciences park Biopôle in Lausanne, Switzerland with laboratory facilities at JLABS @ San Diego, HAYA is led by a world-class team of experts in programmable and precision RNA-targeted therapeutics and is supported by a strong investor consortium. HAYA’s mission is to identify and develop breakthrough disease-modifying therapeutics with the potential for greater efficacy, safety, and accessibility than existing treatments.

Translation-X is a spin-off project at the Biozentrum, University of Basel. We are developing novel pharmacological tools to reduce core symptoms of autism by targeting RNA translational machinery.

Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community

Versameb AG is a privately held biotechnology company focusing on discovering and developing innovative RNA-based drugs for modulation of protein expression, including the ability to simultaneously influence several therapeutic targets, in a controlled manner, with one molecular construct, and cellular targeting. Based in Basel and fully operational from 2018, the company is led by an experienced scientific and leadership team with proven expertise in drug discovery and development from lab bench to patient. Versameb’s proprietary technology platform, VERSagile, optimizes the application of functional RNA in different disease contexts – making RNA druggable in new therapeutic areas others have been unable to solve. The pipeline includes lead candidate programs in Stress Urinary Incontinence (SUI) and solid tumors. Versameb is working towards the completion of a first in-human/proof-of-concept clinical study while advancing its platform.