Engasertib

Biotechs Epicrispr Biotechnologies and Vaderis Therapeutics have raised funds to progress their respective rare disease candidates into pivotal development. Credit: Visual Generation / Shutterstock.com. Two biotech companies, Epicrispr Biotechnologies and Vaderis Therapeutics, have secured fresh capital to take their rare disease assets to late-stage trials – potentially poising these drugs to become the first treatment options in their respective indications. Through a Sanofi Ventures-backed, oversubscribed $90m Series C round, Epicrispr is hoping to push its first-in-class, one-dose epigenetic medicine for progressive inherited muscle wasting disorder, facioscapulohumeral muscular dystrophy (FSHD), down the clinical pipeline to a pivotal study. Epicrispr designed the gene therapy, dubbed EPI-321, to enter a patient’s muscles via a viral vector, where it then suppresses the expression of the mutated DUX4 gene linked to muscle cell death in FSHD. EPI-321 is currently under review in a first-in-human study, where it has already shown favourable early signs of safety, as well as efficacy through significant increases in lean muscle volume and changes in FSHD biomarker levels. If Epicrispr’s therapy were to make it to market, it could become the first drug to secure approval in FSHD – one of the most common forms of muscular dystrophy worldwide. On a global scale, researchers estimate that FSHD impacts one in every 7,500 individuals. Patients with the condition generally experience progressive muscle weakness and wasting in the face, upper arms and shoulder blades. Other therapies in development for FSHD include Avidity Biosciences delpacibart braxlosiran and Arrowhead Pharmaceuticals ARO-DUX4. Novartis acquired Avidity in February 2026 for $12bn. While Epicrispr will siphon a notable portion of the funds towards EPI-321’s development, the California-based biotech will also use some of the cash to expand and accelerate its pipeline, further develop its platform and bolster its manufacturing capacity. Vaderis eyes rare vascular disease market with engasertib Much like Epicrispr, Vaderis is looking to take its lead candidate to pivotal-stage development – eyeing a potential break-out on the market for rare genetic blood vessel condition, hereditary haemorrhagic telangiectasia (HHT), which currently has no specifically approved therapies. By securing $125m in a Goldman Sachs Alternatives and Medixci-backed Series B financing round, Vaderis is looking to begin a global Phase III study on its oral allosteric AKT inhibitor, engasertib – previously called VAD044 – in HHT. According to the biotech, this financing round will support the company’s activities through pivotal clinical development, as well as regulatory submissions and potential US approval. Vaderis secures this cash a little after the biotech published positive long-term extension data on engasertib’s therapeutic potential in HHT, in which the drug diminished the frequency and duration of nosebleeds – a key symptom of HHT. The vascular condition, otherwise known as Osler-Weber-Rendu syndrome, is a rare genetic disorder that causes the blood vessels to form incorrectly. This can cause frequent bleeding episodes, as well as stroke, anaemia and bleeding in the digestive tract. According to GlobalData’s Pharmaceutical Intelligence Center, academic institutions or non-profit organisations are evaluating just under two-thirds of the drugs currently in the clinic for HHT. However, Vaderis could face a potential race to market with other developers such as Alnylam, which is currently conducting a Phase I/II trial (NCT06659640) on its plasminogen-targeting RNA interference (RNAi) therapy, ALN-6400, in HHT. Meanwhile, Diagonal Therapeutics is also testing its bispecific antibody, DIAG723, in the Phase I/II DIAMOND trial (NCT07623525). The biotech designed DIAG723 to restore normal ALK1 signalling, potentially offering as a disease-modifying approach in HHT type 2. GlobalData is the parent company of Pharmaceutical Technology. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

STOCKHOLM, Aug. 11, 2026 /PRNewswire/ -- EQT Life Sciences is investing USD 17.5 million in Vaderis Therapeutics as part of the Swiss company's USD 152 million Series B financing The financing will support the initiation of the global Phase 3 HEROIC study evaluating engasertib, a potential first treatment for hereditary hemorrhagic telangiectasia, a rare genetic vascular disease characterized by severe nosebleeds, chronic anemia, and life-threatening vascular abnormalities, with no approved therapies EQT Life Sciences will draw on its experience supporting biotechnology companies through last-stage clinical development to work alongside the management team in executing the company's development strategy EQT Life Sciences is pleased to announce its participation in Vaderis Therapeutics' USD 152 million Series B financing through one of its managed funds, with a USD 17.5 million investment. The financing was co-led by Life Sciences at Goldman Sachs Alternatives and TCGX, with participation from Omega Funds, Perceptive Advisors, Kalehua Capital and existing investors Medicxi and Droia. The proceeds will support Vaderis with capital needs through potential regulatory approval of engasertib, the company's lead program for hereditary hemorrhagic telangiectasia (HHT). Vaderis Therapeutics is a Swiss clinical-stage biotechnology company developing targeted therapies for rare vascular diseases. Its lead program, engasertib, is being developed as a potential first treatment specifically developed for HHT, a rare inherited blood vessel disorder affecting ~1 in 3,800. HHT causes recurrent and severe nosebleeds (epistaxis), chronic anemia and potentially life-threatening vascular abnormalities for which there are currently no approved therapies. The financing and initiation of the company's global Phase 3 trial, HEROIC, follows publication of positive proof-of-concept and long-term extension data for engasertib in The New England Journal of Medicine, which demonstrated clinically meaningful and sustained improvements across multiple measures of disease in patients with HHT. These data established the scientific foundation for advancing engasertib into pivotal development and its potential to address the significant unmet needs of patients living with HHT. EQT Life Sciences will support Vaderis Therapeutics as it advances engasertib through its Phase 3 development and towards potential regulatory approval. Drawing on its experience supporting biotechnology companies through last-stage clinical development, EQT Life Sciences will work alongside the management team to help execute the company's development strategy. Christoph Broja, CFA, Partner at EQT Life Sciences, who will join the Vaderis board as an observer, said: "HHT remains a serious, lifelong disease with no approved treatment anywhere in the world, despite affecting tens of thousands of patients. Engasertib's proof of concept data gave us real conviction in the science, and we're pleased to support the Vaderis team as they advance engasertib into Phase 3 with the goal of potentially bringing these patients their first dedicated therapy." Azmi Nabulsi, MD, MPH, President and Chief Executive Officer of Vaderis Therapeutics, said: "Today represents a defining moment for HHT patients. Closing this financing and initiating HEROIC as the first Phase 3 study utilizing a molecule specifically developed for HHT marks an exciting new chapter. This milestone reflects the dedication of our patients, investigators, study teams, and advocacy organizations, to whom we extend our deepest gratitude. We are also thankful to our investors for their confidence and support, which have been essential in bringing us to this point." Contact EQT Press Office, [email protected] This information was brought to you by Cision The following files are available for download: 21% more press release views with Request a Demo