Associate Director of Clinical Sciences

Encoded raises $275m for Dravet therapy and other financings. Right after reporting encouraging data with its Dravet syndrome gene therapy, Encoded Therapeutics has raised an impressive $275 million to take the programme into a pivotal trial. At the European Epilepsy Congress (EEC) this week, Encoded showcased results of the phase 1/2 POLARIS trial of ETX101 in Dravet syndrome associated with mutations in the SCN1A gene, including a big reduction in seizure frequency - compared to historical controls - in the 12 months after the one-shot therapy. Dravet syndrome is a severe form of developmental and epileptic encephalopathy that begins in infancy and causes prolonged seizures that do not respond well to current anticonvulsant drugs, as well as behavioural and developmental delays. Treatment with ETX101 was also associated with "encouraging" developmental gains among subjects in POLARIS, according to the South San Francisco company. The new financing, co-led by GV and another unidentified healthcare fund, will also fund the scale-up of manufacturing for ETX101 for forthcoming trials, as well as development of a second drug candidate - codenamed ETX301 - that Encoded has in preclinical testing for post-amputation neuroma pain. The financing was also supported by ARCH Venture Partners, Braidwell LP, Farallon Capital Management, Illumina Ventures, Invus, Janus Henderson Investors, Matrix Capital Management, Nolan Capital, RTW Investments, SoftBank Vision Fund 2, and Venrock. Other recent financing rounds. Also this week, Seattle start-up Brainchild Bio closed a $116 million Series A round earmarked for clinical trials of its CAR-T therapies for central nervous system cancers, headed by BCB-276, a B7-H3-targeted therapy for rare and aggressive paediatric tumour diffuse intrinsic pontine glioma (DIPG). The therapy is currently in the phase 2 ILLUMINATE study, with results due in 2028. An undisclosed private family fund and foundation led the round, with participation from the company's seed investor, Seattle Children's Hospital, and new investor WRF Capital. Meanwhile, south San Francisco-based Moonwalk Biosciences, which specialises in RNA interference drugs for obesity and cardiometabolic diseases, added to its coffers with a $70 million second round co-led by Alpha Wave and YK Bioventures and backed by Eli Lilly, Gaorong Ventures, and existing investors ARCH Venture Partners, Khosla Ventures, and Future Ventures. Armed with the new cash, Moonwalk plans to take its lead drug, MW101 for obesity, into phase 1 testing before the end of next year. The drug, which uses adipose-selective delivery chemistry licensed from China's Suzhou Siran Biotechnology, acts via an undisclosed, non-incretin mechanism that could differentiate it from GLP-1 agonist therapies for weight loss. TwoStep Therapeutics raised $62.5 million in a Series A, providing the resources needed to take its lead peptide-drug conjugate programme, TS-104, into clinical testing for solid tumours. According to San Carlos, California-based TwoStep, TS-104 uses a polyspecific integrin-binding peptide (PIP) that binds to multiple integrins found on solid tumours, delivering a cell-killing MMAE payload. Insight Partners and Medical Excellence Capital acted as co-lead investors, with Merck KGaA's VC arm M Ventures and Pfizer Ventures also taking a prominent role, with existing backers NFX, 2048 Ventures, and Stanford University also participating. Kura Oncology has spun out a new biotech, Caspian Therapeutics, that will develop its menin inhibitor technology outside cancer with the help of $50 million in financing from BVF Partners, supported by Eli Lilly, the T1D Fund, Invus, Montanova Capital, and Kura - which is retaining a half-share in the start-up. Caspian's lead asset is KO-7246, a menin inhibitor currently in preclinical development that could have potential in type 1 and type 2 diabetes and other cardiometabolic disorders by encouraging the expansion of insulin-producing beta cells in the pancreas. The new San Diego biotech said it plans to file for approval to start human trials of KO-7246 "as soon as practicable." Finally this week, New Haven, Connecticut's Cloverleaf Bio has emerged with $33 million in seed financing and a mission to develop a novel class of engineered transfer RNA (tRNA) based payloads for cancer. Its lead programmes - both in preclinical development - are CLB-001, an inhibitory tRNA asset targeting hepatocellular carcinoma, and an antibody-tRNA conjugate (ATC) asset for colorectal cancer. The round was led by 4BIO Capital with backup from AbbVie Ventures, Eli Lilly, and Boehringer Ingelheim Venture Fund, alongside Draper Associates, Mission BioCapital, and American Cancer Society BrightEdge. 11 September, 2026

Encoded raises $275 million to push Dravet gene therapy into pivotal testing. 10 September 2026 US biotech company Encoded Therapeutics has closed a $275 million series F financing that will fund pivotal testing of ETX101, a gene therapy for Dravet syndrome, a severe form of childhood epilepsy. The privately held South San Francisco firm announced the round on Wednesday, days after presenting updated trial data at the European Epilepsy Congress. The financing was co-led by GV, the venture arm of Alphabet (Nasdaq: GOOGL), and a healthcare fund that was not named. Others taking part included ARCH Venture Partners, Braidwell, Farallon Capital Management, Illumina Ventures, Invus, Janus Henderson Investors (NYSE: JHG), Matrix Capital Management, Nolan Capital, RTW Investments, SoftBank Vision Fund 2 and Venrock. This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here. Try before you buy Free. 7 day trial access * All the news that moves the needle in pharma and biotech * Exclusive features, podcasts, interviews, data analyses and commentary from its global network of life sciences reporters. * Receive The Pharma Letter daily news bulletin, free forever. Become a subscriber £820. Or £77 per month * Unfettered access to industry-leading news, commentary and analysis in pharma and biotech. * Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results. * Daily roundup of key events in pharma and biotech. * Monthly in-depth briefings on Boardroom appointments and M&A news. * Choose from a cost-effective annual package or a flexible monthly subscription The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It's part of the key information for keeping me informed Chairman, Sanofi Aventis UK More on this story... 26 August 2026 7 September 2026 Companies featured in this story. Sign up to receive email updates Join industry leaders for a daily roundup of biotech & pharma news Today's issue. 10 September 2026 Company spotlight. A biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. More features in biotechnology. 10 September 2026