The company was testing CAP-002, a gene therapy designed to restore STXBP1 protein levels by crossing the blood-brain barrier.<\/p>\n
iStock.com\/ktsimage<\/p>\n
A gene therapy trial halted after a child\u2019s death, a CRISPR test to detect tuberculosis, a new device for antisense therapies, and more led the news this week.<\/p>\n
Welcome to the Weekly Rundown where the DDN \u00a0editors cover this week\u2019s top biotech and pharma news.<\/p>\n
Capsida pauses rare disease gene therapy after child dies<\/p>\n
On Wednesday, Capsida Biotherapeutics<\/a> announced in a letter<\/a> that the first patient to participate in the SYNRGY trial has passed away. In the letter, Capsida noted that they have paused the trial and are now working to investigate the root cause of the patient\u2019s death. The trial aimed to treat children with a rare developmental encephalopathy caused by mutations in the STXBP1 (syntaxin binding protein 1) gene. The company was testing CAP-002 as a first-in-class gene therapy designed to restore normal STXBP1 protein levels by penetrating through the blood-brain barrier. On their website, the company states that the therapy provides \u201ca superior safety profile compared to traditional gene therapy approaches by lowering dose and detargeting non-therapeutic organs.\u201d The news adds to recent reports<\/a> of three deaths in Sarepta Therapeutics<\/a>\u2019 gene therapy programs, which could lead to greater scrutiny for all gene therapies. \u2013 Allison Whitten<\/p>\n New CRISPR test detects tuberculosis with simple tongue swab<\/p>\n Tuberculosis (TB) is the world\u2019s deadliest infectious disease, affecting more than 10 million people and killing over a million each year, with millions of cases going undiagnosed due to limited testing access. Tulane University researchers have now developed a CRISPR-based TB test that works with a simple tongue swab. Unlike current sputum-based methods, which are invasive, hard to collect, and not always reliable, the new \u201cActCRISPR-TB\u201d assay can detect TB in samples with very low bacterial counts and deliver results in under an hour. In clinical studies published in Nature Communications<\/a>, the test detected TB from tongue swabs more effectively than traditional methods (74 percent versus 56 percent) and showed high accuracy across respiratory, stool, and spinal fluid samples. The painless, non-invasive swabs could enable large-scale screenings, especially in low-resource settings and among patients who cannot produce sputum. \u2013 Bree Foster\u00a0<\/p>\n Biogen bets on new delivery device to advance antisense oligonucleotide therapies<\/p>\n Biogen has agreed<\/a> to acquire Massachusetts-based Alcyone Therapeutics<\/a> for $85 million upfront plus milestones, securing full rights to ThecaFlex DRx<\/a>\u2122, an implantable device being developed for intrathecal delivery of antisense oligonucleotides. The system is designed to provide an alternative to repeated lumbar punctures, with initial studies evaluating use alongside Biogen\u2019s spinal muscular atrophy therapy, nusinersen (Spinraza). The deal will bring Alcyone staff into Biogen\u2019s drug delivery team and position Biogen to lead development, manufacturing, and commercialization of the device, while Alcyone\u2019s remaining assets will spin out into a new company, Neela Therapeutics. \u2013 Andrea Corona<\/p>\n Roche pays up to $3.5 billion for 89bio and its late-stage MASH drug<\/p>\n Roche has agreed to acquire US biotech firm 89bio<\/a>, focusing on its lead drug, pegozafermin. The therapy is a long-acting FGF21 (Fibroblast Growth Factor 21) analogue in late-stage development for moderate to severe Metabolic Dysfunction-Associated Steatohepatitis (MASH), a liver disease commonly associated with obesity. Pegozafermin showed promising results<\/a> in a Phase 2b trial<\/a> of 222 patients with biopsy-confirmed MASH and moderate to severe fibrosis. They improved liver fibrosis by up to 27 percent compared with 7 percent in the placebo group, and achieved MASH resolution in up to 37 percent of patients versus 2 percent for the placebo. Designed with anti-fibrotic and anti-inflammatory mechanisms, the therapy also demonstrated a favorable safety profile, with the most common side effects being nausea and diarrhea. \u201cThis acquisition further strengthens our portfolio in cardiovascular, renal, and metabolic diseases and offers opportunities to explore combinations with existing programmes in our pipeline,\u201d\u00a0said<\/a> Thomas Schinecker<\/a>, Roche Group CEO. The acquisition is expected to close in the fourth quarter of 2025, with a total deal value of up to $3.5 billion, including milestone payments. \u2013 Bree Foster<\/p>\n Arvinas and Pfizer to out-license breast cancer drug<\/p>\n Arvinas<\/a> said<\/a> it will partner with Pfizer to out-license vepdegestrant, an oral proteolysis targeting chimera (PROTAC) estrogen receptor degrader now under FDA review for ESR1 (estrogen receptor 1)-mutated breast cancer, to a third-party commercial partner. The decision shifts Arvinas away from directly marketing the drug while it continues to co-develop with Pfizer. The move may also signal the companies\u2019 interest in reducing the costs and resources required to compete in the crowded breast cancer market while allowing Arvinas to focus on its earlier-stage PROTAC programs. Alongside the update, the company announced additional restructuring measures, including a 15 percent workforce reduction and cost-saving steps expected to deliver more than $100 million in annual savings compared with 2024. Arvinas\u2019 board also authorized a $100 million stock repurchase program and reaffirmed cash runway guidance through the second half of 2028. \u2013\u00a0Andrea Corona\u00a0<\/p>\n Novartis inks $5.7B licensing deal with Monte Rosa Therapeutics\u00a0<\/p>\n For the second time in one year, Novartis has backed Monte Rosa Therapeutics<\/a> on their molecular glue<\/a> degrader-based medicines. The agreement includes a $120 million upfront payment and gives Novartis an exclusive license to an undisclosed discovery target, and provides Monte Rosa scientists with the funding to apply their artificial intelligence (AI)-based product engine called QuEEN to discover and develop degraders for immune-mediated diseases. Novartis\u2019 first deal<\/a> with Monte Rosa came last October with a $150 million licensing agreement for the asset MRT-6160, a molecular glue degrader of VAV1 (vav guanine nucleotide change factor 1). The molecular glue degrader market is forecast to grow significantly<\/a>, rising from $1.2 billion in 2024 to $5.0 billion by 2033. \u201cThis new agreement underscores our commitment to advancing targeted protein degradation as a promising approach to address immune-mediated diseases with high unmet need. We believe Monte Rosa\u2019s QuEEN\u2122 platform has the potential to uncover new insights in this field,\u201d said<\/a> Fiona Marshall<\/a>, President of Biomedical Research at Novartis. \u2013 Allison Whitten<\/p>\n","protected":false},"excerpt":{"rendered":"The company was testing CAP-002, a gene therapy designed to restore STXBP1 protein levels by crossing the blood-brain…\n","protected":false},"author":3,"featured_media":239221,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[26],"tags":[815,159,67,132,68],"class_list":{"0":"post-239220","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-genetics","9":"tag-science","10":"tag-united-states","11":"tag-unitedstates","12":"tag-us"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@us\/115231681591641299","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts\/239220","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/comments?post=239220"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts\/239220\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/media\/239221"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/media?parent=239220"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/categories?post=239220"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/tags?post=239220"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}