Novel Gene Therapy Program Offers a Promising Opportunity for Curative Therapy in DiabetesAUSTIN, Texas, April 29, 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, today announced that its research collaborators have been selected to present at the upcoming American Society of Gene and Cell Therapy's (ASGCT) 28th Annual Meeting being held May 13-17, 2025 i ...

Dublin, April 29, 2025 (GLOBE NEWSWIRE) -- The "Cone Rod Dystrophy Market: Analysis By Type, By Treatment Type, By End User, By Region Size and Trends - Forecast up to 2030" has been added to ResearchAndMarkets.com's offering. Cone rod dystrophy represents a group of genetic eye disorders impacting the retina's cone and rod cells. The market for treating this condition is witnessing substantial growth, projected to rise from a value of US$131.29 million in 2024 to US$177.59 million by 2030. This growth is ...

About Insmed Insmed Incorporated is a people-first global biopharmaceutical company striving to deliver first- and best-in- class therapies to transform the lives of patients facing serious diseases. The Company is advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery focused on serving patient communities where the need is greatest. Insmed's most advanced programs are in pulmonary and inflammatory conditions, including a therapy ap ...

- Oral presentation on tau silencing gene therapy VY1706, which has previously shown up to 73% knockdown of tau mRNA in NHPs in the CNS following a single IV dose of 1.3e13 vg/kg - - Featured data also include anti-amyloid gene therapy for Alzheimer’s disease, as well as multiple presentations on Voyager’s continued enhancements to its highly BBB penetrant novel capsids - LEXINGTON, Mass., April 28, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to lev ...

NEW YORK, April 28, 2025 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today unveils research data on TALEN®-mediated non-viral transgene insertion for advancing cellular and gene therapies, and advancements in genetic editing using TALE base editors (TALEB), at the Society of Gene and Cell Therapy (ASGCT) annual meeting, that will be held ...

BURLINGTON, Mass., April 28, 2025 (GLOBE NEWSWIRE) -- Fractyl Health, Inc. (Nasdaq: GUTS) (“the Company”), a metabolic therapeutics company focused on pattern-breaking approaches to treat the root causes of obesity and type 2 diabetes (T2D), today announced that it will deliver an oral presentation of compelling preclinical data from its Rejuva single-administration Smart GLP-1 pancreatic gene therapy platform at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, taking place May 13- ...

-- PCRX-201 demonstrated two years of clinically meaningful improvements in pain, stiffness and function across all structural severity subgroups, including advanced disease -- -- Study Findings Presented at 2025 OARSI World Congress-- BRISBANE, Calif., April 28, 2025 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to deliver innovative, non-opioid pain therapies to transform the lives of patients, today announced new data demonstrating its locally administ ...

Additional Gene Therapy Technology Expands Genprex's Portfolio of Oncology LicensesAUSTIN, Texas, April 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, today announced it has signed an exclusive patent license agreement with NYU Langone Health related to the drug candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid). The therapy is under investi ...

Summary of Pacira BioSciences Inc (PCRX) Update / Briefing April 27, 2025 Company Overview - **Company**: Pacira BioSciences Inc (PCRX) - **Focus**: Development of innovative therapies for pain management and degenerative diseases, specifically targeting osteoarthritis Key Points - **Commitment to Change**: The company emphasizes its dedication to creating lasting improvements for patients suffering from pain and degenerative diseases like osteoarthritis [1] - **Clinical Development Plan**: Introduction of PCRX201, an innovative gene therapy aimed at addressing both the underlying causes and symptoms of knee osteoarthritis [1] Additional Important Content - **Target Patient Population**: The focus on osteoarthritis indicates a strategic alignment with a significant patient demographic that experiences chronic pain [1]

Core Insights - The FDA has granted Breakthrough Therapy designation to AMT-130 for Huntington's disease, highlighting the urgent need for effective treatments and the promising interim data indicating potential disease progression slowing [1][2] - The designation is supported by clinical data from ongoing Phase I/II trials, with interim data showing dose-dependent slowing of disease progression based on the cUHDRS after 24 months [2] Regulatory Updates - Additional regulatory updates and guidance on the Biologics License Application (BLA) submission for AMT-130 are expected in the second quarter of 2025 [1] - The Breakthrough Therapy designation allows for expedited development and review processes, including intensive guidance on drug development and senior management involvement from the FDA [3] Huntington's Disease Overview - Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies to slow its progression [4] - The disease is characterized by motor symptoms, behavioral abnormalities, and cognitive decline due to a genetic mutation in the huntingtin gene [4] Company Background - uniQure is focused on advancing gene therapies, including AMT-130 for Huntington's disease, and has previously achieved significant milestones with its gene therapy for hemophilia B [5] - The company is developing a pipeline of gene therapies for various severe diseases, including ALS and Fabry disease [5]