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.
