# gene therapies (news sources) — RSS Amplifier Recent posts from the 3 feeds in the RSS Amplifier directory that cover gene therapies. Page: Feed: --- ## [Capricor investor calls for change as company awaits decision on Duchenne candidate](https://www.biospace.com/business/capricor-investor-calls-for-change-as-company-awaits-decision-on-duchenne-candidate) _2026-08-21 · Heather McKenzie · Biopharma Business News_ Kaos Capital in a letter to other Capricor Therapeutics shareholders called for “an immediate meeting, board change, and capital-preservation plan.” The biotech recently put all other pipeline work on pause as it awaits an Aug. 22 PDUFA date for embattled cell therapy deramiocel. ## [Lawmakers urge FDA to enact new China policies after 3 gene therapy deaths](https://www.biospace.com/policy/lawmakers-urge-fda-to-enact-new-china-policies-after-3-gene-therapy-deaths) _2026-08-21 · Gabrielle Masson · Biopharma Business News_ Three fatalities occurring recently in China clinical trials have spurred two members of U.S. Congress to urge the FDA to enact new policies designed to protect American patients. ## [Werewolf and Ambros to tie the knot in $150M rare disease deal](https://www.biospace.com/deals/werewolf-and-ambros-to-tie-the-knot-in-150m-rare-disease-deal) _2026-08-21 · Michael Gibney · Biopharma Business News_ The merger, which would create a new company bearing Ambros’ name, will help support development of Ambros' non-opioid painkiller for patients with debilitating limb injuries. ## [Novo Nordisk starts trialing lower maintenance doses of oral Wegovy](https://www.biospace.com/drug-development/novo-nordisk-starts-trialing-lower-maintenance-doses-of-oral-wegovy) _2026-08-21 · Nick Paul Taylor · Drug Development News_ The Phase 3 study is evaluating two doses of Novo’s Wegovy pill in people with obesity or overweight in combination with a reduced-calorie diet and increased physical activity. ## [Roche commits $750m to double Oregon-based US manufacturing capacity](https://www.pharmaceutical-technology.com/news/roche-750m-oregon-us-injectable-manufacturing-site/) _2026-08-21 · Annabel Kartal Allen · Pharmaceutical Technology_ Roche's Oregon fill-finish site will produce both autoinjectors and pre-filled syringes – serving its pipeline and portfolio. ## [Eli Lilly’s weekly insulin jab Onswik set for NHS use if approved](https://www.pharmaceutical-technology.com/news/eli-lillys-weekly-insulin-jab-onswik-set-for-nhs-use-if-approved/) _2026-08-21 · Robert Barrie · Pharmaceutical Technology_ Lilly’s drug could cut the number of injections each patient needs by 85%. ## [WellSpan and Johns Hopkins renew partnership for advanced cancer care access](https://www.pharmaceutical-technology.com/news/wellspan-johns-hopkins-advanced-cancer-care/) _2026-08-21 · Salong Debbarma · Pharmaceutical Technology_ WellSpan Health and Johns Hopkins Medicine have renewed their oncology collaboration to widen access to advanced care throughout central Pennsylvania, US. ## [Charles River and MVC to develop enterovirus vaccine](https://www.pharmaceutical-technology.com/news/charles-river-mvc-enterovirus-vaccine/) _2026-08-21 · Salong Debbarma · Pharmaceutical Technology_ Charles River Laboratories International and MVC have entered a collaboration to support the characterisation, along with validation of the virus seed bank for the latter’s next-generation multivalent enterovirus vaccine. ## [Roche pours $750M into Oregon manufacturing plant as obesity push expands](https://www.biospace.com/drug-delivery/roche-pours-750m-into-oregon-manufacturing-plant-as-obesity-push-expands) _2026-08-20 · Gabrielle Masson · Biopharma Business News_ After committing $2 billion to a new North Carolina facility to boost capacity for its next-gen obesity candidates, Roche's Genentech is putting down more cash to expand a device fill-finish site in Oregon. ## [Scribe’s early-stage IPO standout defies biotech’s derisking trend](https://www.biospace.com/business/scribes-early-stage-ipo-standout-defies-biotechs-derisking-trend) _2026-08-20 · Gabrielle Masson · Biopharma Business News_ Scribe Therapeutics’ one-time epigenetic treatment is designed to slash the adherence burden of chronic cholesterol-lowering therapies while sidestepping the irreversibility of gene editing. ## [profullstack on Ko‑fi (Sponsored)](https://crawlproof.com/a/sMPCQy1enF79) _2026-08-20 · **Sponsored**_ Open the creator's Ko‑fi page. ## [Regeneron wins FDA approval to challenge Ipsen for rare bone disease market](https://www.biospace.com/fda/regeneron-wins-fda-approval-to-challenge-ipsen-for-rare-bone-disease-market) _2026-08-20 · Nick Paul Taylor · Biopharma Business News_ Regeneron’s Pasatru is the second fibrodysplasia ossificans progressiva drug approved in the U.S., following the 2023 approval of Ipsen’s Sohonos. ## [Ultragenyx bags first gene therapy approval as previously rejected Sanfilippo asset awaits decision](https://www.biospace.com/fda/ultragenyx-bags-first-gene-therapy-approval-as-previously-rejected-sanfilippo-asset-awaits-decision) _2026-08-20 · Nick Paul Taylor · Biopharma Business News_ The accelerated greenlight for Ultragenyx’s gene therapy for glycogen storage disease has raised analyst expectations for approval of UX111, which the FDA rejected last summer and is currently reviewing for a second time. A decision is expected by mid-September. ## [Merck’s Moderna-partnered vaccine success clears view of post-Keytruda future](https://www.biospace.com/business/mercks-moderna-partnered-vaccine-success-clears-view-of-post-keytruda-future) _2026-08-20 · Annalee Armstrong · Biopharma Business News_ Investors had been pessimistic about the probability of success for the personalized cancer vaccine intismeran autogene, so the Phase 3 win was a welcome surprise. ## [Rewriting the rules of CMV prevention](https://www.biospace.com/drug-development/rewriting-the-rules-of-cmv-prevention) _2026-08-20 · Jennifer C. Smith-Parker · Drug Development News_ In this episode of Denatured, you’ll hear from Ian McGowan, Chief Medical Officer at Synklino and James Hunter, associate professor of transplantation at Oxford University. We explore transplantation and antiviral innovation and why CMV remains one of the most persistent and consequential challenges facing transplant patients today. ## [Regeneron’s Pasatru wins FDA approval in ultra-rare ‘stone man disease’](https://www.pharmaceutical-technology.com/news/regenerons-pasatru-wins-fda-approval-in-ultra-rare-stone-man-disease/) _2026-08-20 · Robert Barrie · Pharmaceutical Technology_ Pasatru demonstrated strong efficacy in a Phase III trial, helping patients reduce lesions and flare-ups. ## [Kynexis pockets €97m to advance cognition-focused schizophrenia drug](https://www.pharmaceutical-technology.com/news/kynexis-97m-cognitive-focused-schizophrenia-drug-progression/) _2026-08-20 · Annabel Kartal Allen · Pharmaceutical Technology_ Kynexis will now prepare for registrational studies on lead asset, KYN-5356 in cognitive impairment associated with schizophrenia (CIAS). ## [FDA grants accelerated approval to Ultragenyx’s Genglycos gene therapy](https://www.pharmaceutical-technology.com/news/fda-approval-ultragenyx-genglycos-gene-therapy/) _2026-08-20 · Salong Debbarma · Pharmaceutical Technology_ Ultragenyx Pharmaceutical has received accelerated approval from the FDA for its gene therapy, Genglycos, to treat GSDIa. ## [SandboxAQ introduces AQPotency for virtual drug screening](https://www.pharmaceutical-technology.com/news/sandboxaq-aqpotency-virtual-drug-screening/) _2026-08-20 · Salong Debbarma · Pharmaceutical Technology_ SandboxAQ has introduced AQPotency, a LQM designed to virtually screen and predict the potential of drug candidates without the need for a solved protein structure. ## [7 biopharma M&A deals where layoffs followed](https://www.biospace.com/job-trends/7-biopharma-m-a-deals-where-layoffs-followed) _2026-08-20 · Angela Gabriel · Biopharma Business News_ Workforce cuts this year at seven companies involved in recent mergers and acquisitions will cost over 1,300 employees their jobs. Affected businesses include Arcellx and Tourmaline Bio, acquired by Gilead Sciences and Novartis, respectively. ## [Out of sight, out of work? What’s driving remote job cuts](https://www.biospace.com/job-trends/out-of-sight-out-of-work-whats-driving-remote-job-cuts) _2026-08-20 · Angela Gabriel · Biopharma Business News_ Biopharma professionals sometimes find themselves out of jobs when employers want to bring staff back into the office, as recently seen at biotech EMD Serono and Exelixis. ## [Countdown to Getting Paid (Sponsored)](https://crawlproof.com/a/6HnAyw1bcl3g) _2026-08-20 · **Sponsored**_ Create a shareable wallet timer — deposits buy back time and pay debt down ## [Why pediatric rare oncology outperforms in a volatile biotech market](https://www.biospace.com/drug-development/opinion-why-pediatric-rare-oncology-outperforms-in-a-volatile-biotech-market) _2026-08-20 · Paul Romness · Drug Development News_ Real progress happens where unmet medical need is greatest and patient impact can be clearly measured. Pediatric rare cancers exemplify these criteria and have therefore generated continued investment, even in a capital-constrained environment. ## [BioMarin spends up to $490m for Alesta’s bone disease candidate](https://www.pharmaceutical-technology.com/news/biomarin-spends-up-to-490m-for-alestas-bone-disease-candidate/) _2026-08-19 · Robert Barrie · Pharmaceutical Technology_ The deal provides BioMarin with a potential rival to AstraZeneca’s blockbuster Strensiq in hypophosphatasia. ## [Kolon TissueGene cuts 37 US staffers after Phase 3 fail](https://www.biospace.com/biospace-layoff-tracker) _2026-08-19 · BioSpace Editorial Staff · Biopharma Business News_ Follow along as BioSpace tracks job cuts and restructuring initiatives. ## [Moderna stock nearly doubles as Merck-partnered mRNA cancer vaccine meets Phase 3 goal](https://www.biospace.com/drug-development/moderna-stock-nearly-doubles-as-merck-partnered-mrna-cancer-vaccine-meets-phase-3-goal) _2026-08-19 · Nick Paul Taylor · Drug Development News_ Melanoma was slower to recur in patients receiving Merck and Moderna's mRNA vaccine with Keytruda, supporting use of the combination in adjuvant treatment. The partners plan to take the data to the FDA. ## [Enveda’s exercise-simulating obesity tablet clears early clinical test](https://www.biospace.com/drug-development/envedas-exercise-simulating-obesity-tablet-clears-early-clinical-test) _2026-08-19 · Nick Paul Taylor · Drug Development News_ Touting an “exceptional” gastrointestinal safety profile for its oral obesity drug candidate in a study of healthy volunteers, Enveda plans to test the asset in people stopping GLP-1 treatment. ## [2026 IPO stock tracker: How newly public biotechs are performing](https://www.biospace.com/business/2026-ipo-stock-tracker-how-biotechs-new-to-public-markets-are-performing) _2026-08-19 · Gabrielle Masson · Biopharma Business News_ BioSpace is launching an IPO dashboard to help the industry keep tabs on U.S. biotechs that have taken the public plunge in 2026. ## [Amylyx’s redemption story, Summit’s survival woes, Definium’s anxiety win, more](https://www.biospace.com/fda/amylyxs-redemption-story-summits-survival-woes-definiums-anxiety-win-more) _2026-08-19 · Jef Akst · Biopharma Business News_ Amylyx shares good news for its investigational GLP-1 blocker; Definium announces positive data for its LSD-based therapy in anxiety; Summit/Akeso’s ivonescimab shows degrading survival benefits; patients treated with Neurocrine’s Prader-Willi drug experience serious adverse events; and Eli Lilly cracks down on the black market for next-gen obesity asset retatrutide. ## [Trump expected to nominate Heidi Overton as new FDA commissioner](https://www.pharmaceutical-technology.com/news/trump-nominates-heidi-overton-fda-commissioner/) _2026-08-19 · Annabel Kartal Allen · Pharmaceutical Technology_ Overton could take the helm at the FDA during a tumultuous period for the agency, defined by several resignations, firings and redundancies. ## [Andelyn Biosciences to develop GM2 gangliosidosis therapy](https://www.pharmaceutical-technology.com/news/andelyn-to-develop-gm2-gangliosidosis-therapy/) _2026-08-19 · Salong Debbarma · Pharmaceutical Technology_ Andelyn Biosciences is set to develop and manufacture a gene therapy known as AAV9-GM2, for Queen’s University to treat GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases. ## [BeOne Medicines and partners renew Brukinsa access programme for CLL](https://www.pharmaceutical-technology.com/news/beone-medicines-partners-renew-brukinsa-programme/) _2026-08-19 · Salong Debbarma · Pharmaceutical Technology_ BeOne Medicines, the BeOne Care Foundation, and The Max Foundation have extended their Brukinsa access programme for CLL until 2028. ## [Fast, secure international transfers (Sponsored)](https://crawlproof.com/a/SFCfZMSIPOEZ) _2026-08-19 · **Sponsored**_ Bank transfer, cash pickup, mobile wallet — low fees and real-time tracking ## [Reformed pharmaceutical regulation across India’s drug landscape](https://www.pharmaceutical-technology.com/analyst-comment/reformed-pharmaceutical-regulation-across-india-drug-landscape/) _2026-08-18 · GlobalData Healthcare · Pharmaceutical Technology_ One of the major changes made to India’s primary legal framework for drug price regulation affects the pricing of essential drugs. ## [Updated UK guidance for finerenone introduces new treatment for HF patients](https://www.pharmaceutical-technology.com/analyst-comment/updated-uk-guidance-finerenone-hf/) _2026-08-18 · GlobalData Healthcare · Pharmaceutical Technology_ New guidance from NICE provide a framework for UK care providers using finerenone to manage the treatment of certain types of heart failure.