Event will feature Aravindhan Veerapandiyan, M.D., principal investigator of the AFFINITY DUCHENNE® trialROCKVILLE, Md., June 2, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss new interim functional data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, the company's next-generation investigational gene therapy for the treatment of Duchenne muscular dystrophy. The webcast will feature AFFINITY DUCHENNE principal investigator Aravindhan Veera ...

~ Alignment with FDA continues to support Accelerated Approval pathway ~ ~ BLA submission planned for first quarter of 2026 ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 02, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington’s disease. Following recent Typ ...

Shares of Intellia Therapeutics (NTLA) were down 22.9% yesterday after it announced an update from the ongoing phase III study, evaluating the investigational in vivo genome-editing candidate, nexiguran ziclumeran (nex-z), for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM).Per the company, one of the patients in the phase III MAGNITUDE study, which evaluated nex-z for treating ATTR-CM, experienced grade 4 liver transaminase elevations, indicating a notable increase in liver enzymes. The patient was ...

Group 1 - The article discusses Taysha Gene Therapies, Inc. (NASDAQ: TSHA) and its recent developments, particularly focusing on initial data related to Rett Syndrome and upcoming catalysts in 2024 [2]. - The author operates the Biotech Analysis Central service, which provides in-depth analysis of various pharmaceutical companies and includes a library of over 600 biotech investing articles [2]. - The service offers a model portfolio of more than 10 small and mid-cap stocks, along with live chat and analysis to assist healthcare investors in making informed decisions [2].

Taysha Gene Therapies Inc. TSHA on Wednesday announced details of its planned pivotal Part B trial design for TSHA-102 following written alignment from the U.S. Food and Drug Administration (FDA).Single-arm, open-label trial with patients serving as their control (intend N=15).Enrollment of females in the developmental plateau population of Rett syndrome (≥ 6 years).The primary endpoint will assess developmental milestone gain or regain.The safety of TSHA-102 will be evaluated in females in the pre-developm ...

Bio-Techne’s (TECH) GMP reagents, including GMP proteins and small molecules, remain a cornerstone of its cell therapy offering. Acquisitions have continued to play an important role in the company’s efforts to expand its portfolio, as well as enter adjacent markets. Further, the company has begun to pursue clinical diagnostic opportunities on Ella, its automated multiplexing immunoassay instrument platform. However, ongoing macroeconomic volatilities and a challenging research landscape may adversely weigh ...

Shares of Rocket Pharmaceuticals (RCKT) plunged 62.8% on Tuesday after the company announced a patient death in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating patients with Danon disease.The company confirmed that one of the patients in the study experienced an acute systemic infection, an unexpected serious adverse event, which resulted in his untimely death.Following the turn of events, RCKT has voluntarily paused further dosing in the study evaluatin ...

Company Overview - Insmed Incorporated is a global biopharmaceutical company focused on delivering first- and best-in-class therapies for patients with serious diseases [3][4] - The company is advancing a diverse portfolio of approved and investigational medicines, particularly in pulmonary and inflammatory conditions [3] - Insmed's early-stage programs include various technologies such as gene therapy, AI-driven protein engineering, and synthetic rescue [3] Upcoming Event - Management will present at the 2025 Goldman Sachs Annual Global Healthcare Conference on June 11, 2025, at 9:20 a.m. ET [1] - The event will be webcast live and available for 30 days post-event on the company's investor relations website [2]

Novel Gene Therapy is Addressing Both Type 1 and Type 2 Diabetes AUSTIN, Texas, May 28, 2025 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, announced that its research collaborators presented positive preclinical data and research from studies of GPX-002, the Company's diabetes gene therapy drug candidate, in an oral presentation at the American Society of Ge ...

Company Overview - uniQure N.V. is a gene therapy company with four clinical programs and several data catalysts expected in 2025 and 2026 [1] - The flagship candidate of the company is AMT-130, which has received several FDA designations including Breakthrough Therapy, RMAT, Fast-Track, and Orphan Drug [1] Clinical Programs - The company is actively working on its clinical programs, with a focus on advancing its lead candidate AMT-130 [1]