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Published on 04:12 GMT

Commercial Bottlenecks in Gene Therapy Development

Gene therapy has shown remarkable potential to provide transformative treatment effects, but unfortunately the financial costs associated with many gene therapy products hinder efforts for continued innovation and accessibility of the products to patients. …

Source: CGTLive - Neutral
Published on 03:13 GMT

Nicholas Giovannone, PhD, on the Potential Benefits of Redosing Gene Therapy

This is the second part of an interview with Nicholas Giovannone, PhD. For the first part, click here. “Our hope broadly is that this can expand treatment options for patients and improve overall for the field the efficacy of gene therapies and durability …

Source: CGTLive - Neutral
Published on May 17, 2025

Brian Kim, MBA, on Multiomic Single Cell Sequencing and the Ebbs and Flows of the Gene Therapy Field

“I think this is an interesting time we're living in right now and cell and gene therapy is going through a transition period right now, but I think it's really important to note that it's a transition period. I don't think people should …

Source: CGTLive - Neutral
Published on May 17, 2025

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on the Implications of the First Successful ...

This is the third part of an interview with Kiran Musunuru, MD, PhD and Rebecca Ahrens-Nicklas, MD, PhD. For the first part, click here. For the second part, click here. “It's really moving medicine to be more like surgery in a way—more like a type of …

Source: CGTLive - Neutral
Published on May 17, 2025

Rocket’s PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy RP-A601 Improves or Stabilizes Heart Function in Phase 1 Trial

Rocket Pharmaceuticals’ RP-A601, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat plakophilin-2 related Arrhythmogenic Cardiomyopathy (PKP2-ACM), has demonstrated the ability to stabilize or improve heart function …

Source: CGTLive - Neutral
Published on May 17, 2025

Ben Samelson-Jones, MD, PhD, on Conservative Management Following Treatment With DMD Gene Therapy Elevidys

“I think the big picture take-home is that AAV has a set of evolving and unique toxicities, and the clinical care of AAV recipients requires collaboration and developing special specialty expertise. I think that's really important to safely administer …

Source: CGTLive - Neutral
Published on May 17, 2025

Ochsner Transplant Institute's Kidney Program Achieves ELITE Status

Newswise — NEW ORLEANS – Ochsner Health proudly shares its kidney transplant program, part of the Ochsner Transplant Institute, has been awarded ELITE Status within the credentialed Programs of Excellence transplant network by INTERLINK COE Networks & …

Source: Newswise - Neutral
Published on May 16, 2025

Coave Therapeutics Launches CNS-Targeted Gene Therapy Capsid Developed with Proprietary Platform

In a poster presentation at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, held May 13–17, 2025 in New Orleans, Paris-based Coave Therapeutics presented breakthrough data from studies of a novel, central nervous system (CNS)- …

Source: BioPharm International - Pending
Published on May 16, 2025

Shaving, service and success

My previous blogs have looked at other industries, such as airlines, hospitality, and even toy companies, as examples of outstanding customer service. We can learn from these companies and often implement what these successful industries do into our …

Source: Physicians Practice - Neutral
Published on May 16, 2025

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on the Next Steps for Personalized Gene ...

This is the second part of an interview with Kiran Musunuru, MD, PhD and Rebecca Ahrens-Nicklas, MD, PhD. For the first part, click here. “I think the long-term goal... is we want this to be a platform that will benefit many patients. We can do n-of-1 …

Source: CGTLive - Neutral
Published on May 16, 2025

Roger Hajjar, MD, on Helping to Bring Preclinical Gene Therapy Research to First-In-Human Trials

“That's really been the mission and the differentiator for the Institute: to really bring together a set of platforms to help our investigators get to first-in-human [trials] because overall, the mission of the academic medical center that we're in …

Source: CGTLive - Neutral
Published on May 15, 2025

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on Treating CPS1 Deficiency With ...

“Something worth emphasizing again is that this was a truly personalized therapy... This drug was made for one patient, this one kid with this very, very, bad urea cycle disorder, and it's tuned to his mutation to correct his mutation. As such, this …

Source: CGTLive - Neutral
Published on May 15, 2025

Early Data Show Potential of Tevard’s tRNA Therapy in Duchenne Muscular Dystrophy

In an oral presentation on day three of the 2025 American Society of Gene & Cell Therapy Annual Meeting (ASGCT 2025)—held May 13-17 in New Orleans—the privately held biotechnology company Tevard Biosciences presented preclinical data indicating that …

Source: BioPharm International - Pending
Published on May 15, 2025

EG 427 Presents HSV-1 Tech for Neurology at ASGCT 2025

The France-based genetic medicines developer EG 427 has provided details on its two posters exhibited on the first night of the 28th annual meeting of the American Society of Gene and Cell Therapy (ASGCT), running from May 13–17, 2025 in New Orleans (1). …

Source: BioPharm International - Pending
Published on May 15, 2025

World's first patient treated with personalized CRISPR therapy

CHOP’s Rebecca Ahrens-Nicklas, MD, PhD, and Penn Medicine’s Kiran Musunuru, MD, PhD. Credit: CHOP PHILADELPHIA & NEW ORLEANS – In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a …

Distribution channels: Healthcare & Pharmaceuticals Industry
Published on May 17, 2025

STAT+: At gene therapy meeting, no one wants to talk about the Vinay Prasad (not) in the room

NEW ORLEANS — Last year, hundreds of people poured in to see Peter Marks, the Food and Drug Administration’s head of biologics, speak at the American Society of Gene and Cell Therapy conference. The regulator was seen by many advocates, researchers, and …

Source: STAT - Massachusetts - Neutral
Published on May 16, 2025

CAMP4 Presents Translational Data from SYNGAP1-Related Disorders Program Showcasing Increased Protein in Non-Human ...

Haploinsufficient SYNGAP1 mice treated with CMP-SYNGAP-01 demonstrated an increase in SYNGAP1 protein levels; treatment rescued multiple SYNGAP1-dependent behavioral phenotypes CMP-SYNGAP-01 administration led to a significant increase in SYNGAP1 protein …

Distribution channels: Culture, Society & Lifestyle, Healthcare & Pharmaceuticals Industry ...
Published on May 17, 2025

STAT+: Day 3 at ASGCT: Vinay Prasad speculation, screening newborns, promising data

NEW ORLEANS — Greetings from the home stretch of American Society of Gene and Cell Therapy conference, where everyone is still talking about Baby KJ and no one wants to talk about Vinay Prasad. The Vinay Prasad in the room Last year, hundreds of people …

Source: STAT - Massachusetts - Neutral
Published on May 15, 2025

Nicholas Giovannone, PhD, on Using Bispecific Antibodies to Redose AAV Gene Therapy

“The end result was that we were able to redose with a second AAV vector in the monkeys and achieved much higher levels of transduction than monkeys that received no immunomodulation.” Adeno-associated virus (AAV) vector-based approaches are very common in …

Source: CGTLive - Neutral

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Published on May 17, 2025 - 23:56 GMT

Mental Health Crisis Among U.S. Teens Deepens as Many Remain Untreated

Many U.S. teens face mental health struggles without support, Key Healthcare urges better access to residential care and awareness nationwide. CA, UNITED STATES, May 17, 2025 /⁨EINPresswire.com⁩/ -- New national data reveals a concerning gap in …

Distribution channels: Healthcare & Pharmaceuticals Industry ...
Published on May 16, 2025 - 17:34 GMT

Effective Practice Management Unveils 'New Patient Module' to Combat Healthcare Practice Revenue Gaps

New coaching program solves the critical patient acquisition gap plaguing holistic practices. CLEARWATER, FL, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- Effective Practice Management® is proud to announce the launch of its New Patient …

Distribution channels: Business & Economy, Culture, Society & Lifestyle ...

Published on May 16, 2025 - 17:26 GMT

Dr. Peter A. McCullough selected as Top Healthcare Freedom Advocate by IAOTP

Dr. Peter A. McCullough will be honored at the International Association of Top Professionals annual awards gala in Las Vegas NEW YORK, NY, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- Dr. Peter A. McCullough, World Renowned Doctor, Public …

Distribution channels: Business & Economy, Emergency Services ...
Published on May 16, 2025 - 14:56 GMT

New Research Reveals 45% of Excluded Healthcare Providers Concentrated in Just 5 States Creating Hidden Compliance Risks

Comprehensive analysis of 7,212 excluded providers identifies geographic risk patterns that traditional compliance programs miss SAN FRANCISCO, CA, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- Assured, an NCQA-certified credentials verification …

Distribution channels: Healthcare & Pharmaceuticals Industry, IT Industry ...

Published on May 16, 2025 - 13:23 GMT

Ketorolac Market Key Players Analysis -Allergan plc, Deepcare Health Pvt Ltd, Cadila Healthcare, Intas Pharmaceuticals

BURLINGAME, CA, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- Qualitative latest Research report (2025-2032) on the Ketorolac Market by Coherent Market Insights Provides a deep dive into key market trends, drivers, challenges, and the competitive …

Distribution channels: Healthcare & Pharmaceuticals Industry
Published on May 16, 2025 - 13:15 GMT

GRI Bio to Present at A.G.P.'s Annual Healthcare Company Showcase

Live webcast fireside chat with Marc Hertz, PhD, Chief Executive Officer on Wednesday, May 21st at 2:40 PM ET LA JOLLA, CA, May 16, 2025 (GLOBE NEWSWIRE) -- GRI Bio, Inc. (NASDAQ: GRI) (“GRI Bio” or the “Company”), a biotechnology company advancing an …

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Published on May 16, 2025 - 13:15 GMT

Mercurity Fintech’s Subsidiary Grows Cross-Border Business Advisory Services with New Asia-Pacific Healthcare Client ...

New York, NY, May 16, 2025 (GLOBE NEWSWIRE) -- Mercurity Fintech Holding Inc. (the “Company,” “we,” “us,” “our company,” or “MFH”) (NASDAQ: MFH), a digital fintech group, today announced that its wholly-owned subsidiary, Chaince Securities, Inc. (“Chaince …

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Published on May 16, 2025 - 11:30 GMT

Quantum BioPharma to Attend Sidoti Micro-Cap Investor Conference and the AGP Virtual Healthcare Conference

TORONTO, May 16, 2025 (GLOBE NEWSWIRE) -- Quantum BioPharma Ltd. (NASDAQ: QNTM) (CSE: QNTM) (FRA: 0K91) “Quantum BioPharma” or the “Company”) a biopharmaceutical company dedicated to building a portfolio of innovative assets and biotech solutions, today …

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Healthcare & Pharmaceuticals Industry Press Releases

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Published on May 18, 2025 - 12:00 GMT

Cytokinetics Presents Additional Data Related to Aficamten at the European Society of Cardiology Heart Failure 2025 ...

Two New Analyses from SEQUOIA-HCM on Effect of Aficamten Between Patients with Mild and Moderate-to-Severe Symptoms, and Across Geographic Regions HEOR Analyses of Real-World Data Reveal Disparities in Outcomes In Females and Older Patients with Non- …

Distribution channels: Culture, Society & Lifestyle, Healthcare & Pharmaceuticals Industry ...
Published on May 17, 2025 - 23:56 GMT

Mental Health Crisis Among U.S. Teens Deepens as Many Remain Untreated

Many U.S. teens face mental health struggles without support, Key Healthcare urges better access to residential care and awareness nationwide. CA, UNITED STATES, May 17, 2025 /⁨EINPresswire.com⁩/ -- New national data reveals a concerning gap in …

Distribution channels: Healthcare & Pharmaceuticals Industry ...
Published on May 18, 2025 - 11:28 GMT

Applied Therapeutics Presents Full 12-Month Clinical Results and New Topline Data from INSPIRE Phase 2/3 Trial of ...

Slowing of progression of disease observed via MRI at 24 months Improvements in outcomes and reduction in sorbitol maintained through 24 months Statistically significant correlation between absolute reduction in sorbitol and change in 10MWRT and CMT-FOM …

Distribution channels: Healthcare & Pharmaceuticals Industry ...
Published on May 18, 2025 - 11:25 GMT

Intellia Announces Positive Two-Year Follow-Up Data from Ongoing Phase 1 Study of Nexiguran Ziclumeran (nex-z), in ...

Deep, durable and consistent reductions in TTR were sustained at two years, following a one-time dose of nex-z Clinically meaningful improvements in ATTRv-PN related outcomes observed at 24 months compared to baseline, including in patients who were …

Distribution channels: Healthcare & Pharmaceuticals Industry, Technology ...
Published on May 17, 2025 - 22:57 GMT

Sanctuary Cosmetic Center Celebrates 10 Years of Excellence in Aesthetic Medicine in Tysons Corner, Virginia

Sanctuary Cosmetic Center marks 10 years in Tysons Corner as a top destination for surgical and non-surgical aesthetics, led by Dr. Soheila Rostami. TYSONS CORNER, VA, UNITED STATES, May 17, 2025 /⁨EINPresswire.com⁩/ -- Sanctuary Cosmetic Center, …

Distribution channels: Beauty & Hair Care, Business & Economy ...
Published on May 17, 2025 - 22:46 GMT

British Patients Flock to Türkiye for Affordable Dental Treatments

With dental costs reaching record levels in the UK, more British citizens are travelling to Türkiye for top-quality yet affordable dental care. ANTALYA, TURKEY, May 17, 2025 /⁨EINPresswire.com⁩/ -- With dental costs reaching record levels in the …

Distribution channels: Business & Economy, Companies ...
Published on May 17, 2025 - 22:38 GMT

NBA Champion Kendrick Perkins Joins Cal Dental USA & First Choice Practice Sales at CDA 2025

NBA Champion Kendrick Perkins visits CDA 2025 Day 2, joining Cal Dental USA & First Choice Practice Sales to spotlight affordable, community-driven dental care. LOS ANGELES, CA, UNITED STATES, May 17, 2025 /⁨EINPresswire.com⁩/ -- In a headline-making …

Distribution channels: Healthcare & Pharmaceuticals Industry ...
Published on May 17, 2025 - 22:24 GMT

Batth Medicals Enhances Ultrasound Scanning Standards in Romford, London

ROMFORD, GREATER LONDON, UNITED KINGDOM, May 17, 2025 /⁨EINPresswire.com⁩/ -- A growing demand for more accurate and timely diagnostic services has prompted significant improvements in ultrasound scanning at Batth Medicals, a healthcare clinic in …

Distribution channels: Companies, Healthcare & Pharmaceuticals Industry ...
Published on May 17, 2025 - 12:00 GMT

The Good Feet Store Opens in Geneva to Help Local Residents and Workforce Live the Life They Love

The Leading Retailer of Premium, Personally-Fitted Arch Supports Expands with the Sixth Store in the Greater Chicago Area GENEVA, IL, UNITED STATES, May 17, 2025 /⁨EINPresswire.com⁩/ -- The Good Feet Store, recognized nationally as the market- …

Distribution channels: Business & Economy, Companies ...
Published on May 17, 2025 - 14:40 GMT

Tectonic Therapeutic Presents Complete Results for Positive Phase 1b Clinical Trial of TX45 in Patients with Group 2 ...

Data confirmed TX45’s tolerability profile and improvements in left ventricular function and pulmonary hemodynamics in patients with Group 2 Pulmonary Hypertension in Heart Failure with preserved Ejection Fraction (“PH-HFpEF”) In new echocardiographic …

Distribution channels: Culture, Society & Lifestyle, Healthcare & Pharmaceuticals Industry ...
Published on May 17, 2025 - 13:05 GMT

Brain Cancer Canada Awards $68,000 Research Grant for Clinical Trial of Anti-Cancer Vaccine Targeting DIPG, the Most ...

TORONTO, May 17, 2025 (GLOBE NEWSWIRE) -- Today, on DIPG Awareness Day, Brain Cancer Canada is pleased to award $68,000 for an innovative research initiative led by Dr. Aru Narendran, MD, PhD, and Dr. Patrick Sipila, PhD, at The University of Calgary and …

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Published on May 17, 2025 - 11:15 GMT

Fractyl Health Unveils New Rejuva® Smart GLP-1™ Pancreatic Gene Therapy Preclinical Data Highlighting Durable Potency ...

Data suggests that single dose of RJVA-001 leads to durable metabolic improvements with low systemic GLP-1 exposure in db/db mouse model of T2D— potentially simultaneously addressing durability, adherence, and tolerability challenges seen with current …

Distribution channels: Healthcare & Pharmaceuticals Industry, Media, Advertising & PR ...
Published on May 16, 2025 - 20:47 GMT

The Partnership to End HIV, STDs, and Hepatitis Urges Congress to Reject Medicaid Cuts and Misguided Work Requirements

WASHINGTON, DC, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- The Partnership to End HIV, STDs, and Hepatitis —a coalition including NMAC, The AIDS Institute, National Coalition of STD Directors, NASTAD (National Alliance of State and …

Distribution channels: Business & Economy, Healthcare & Pharmaceuticals Industry ...
Published on May 16, 2025 - 20:37 GMT

Online Dispensary Area 52 Becomes First to Ship to Texas

Area 52 becomes first dispensary to ship to Texas – without a medical card – offering same day shipping for THC gummies, THCA flower, and delta 9 products. AUSTIN, TX, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- Area 52 has announced the …

Distribution channels: Companies, Consumer Goods ...
Published on May 16, 2025 - 19:51 GMT

Oatley Vigmond LLP Acclaimed in the 2025 CL Top Personal Injury Boutiques

BARRIE, ONTARIO, CANADA, May 16, 2025 /⁨EINPresswire.com⁩/ -- Canadian Lawyer conducted a far-reaching analysis of the legal industry and is proud to recognize Oatley Vigmond LLP as one of the standout performers and celebrate their exemplary …

Distribution channels: Healthcare & Pharmaceuticals Industry, Insurance Industry ...
Published on May 16, 2025 - 19:32 GMT

Rehabilitation Hospital of Indiana (RHI) Sports Program Receives $30,000 Grant from The Hartford

Company also surprises local beep baseball team with custom equipment INDIANAPOLIS, IN, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- RHI Sports, a non-profit organization and primary provider of adaptive sports opportunities for individuals …

Distribution channels: Companies, Culture, Society & Lifestyle ...
Published on May 16, 2025 - 19:25 GMT

RIIG - HOOTL Secures Strategic Investment in UAE

Relationship Expands Middle East Regional Footprint, Rebrands to RIIG-HOOTL CHARLOTTESVILLE, VA, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- RIIG – HOOTL, a risk intelligence and cybersecurity solutions provider, is proud to announce the …

Distribution channels: Banking, Finance & Investment Industry, Business & Economy ...
Published on May 16, 2025 - 19:25 GMT

Beyond PE: Maitland FL Private Gym Darwin Fitness Helps Homeschooled Kids Stay Fit With Structured Daytime Training

Darwin Fitness Launches Homeschool Personal Training Program to Enhance Physical and Cognitive Development in Children WINTER PARK, FL, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- Darwin Fitness, a youth-focused private training gym …

Distribution channels: Education, Healthcare & Pharmaceuticals Industry ...
Published on May 16, 2025 - 19:23 GMT

ScribeEMR to Provide Rural Health AI Medical Scribing & Coding Solutions at NRHA’s 48th Annual Rural Health Conference

WOBURN, MA, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- ScribeEMR, a leading provider of AI-powered healthcare documentation solutions, virtual scribing, coding and medical office services, will exhibit at the National Rural Health …

Distribution channels: Companies, Healthcare & Pharmaceuticals Industry ...
Published on May 16, 2025 - 18:47 GMT

Cincinnati Massage for Mental Health Pioneers Trauma-Informed Bodywork Approach

CINCINNATI, OH, UNITED STATES, May 16, 2025 /⁨EINPresswire.com⁩/ -- Cincinnati Massage for Mental Health (CMMH) is redefining the intersection of massage therapy and mental health care through its innovative, trauma-informed approach to healing. …

Distribution channels: Beauty & Hair Care, Healthcare & Pharmaceuticals Industry ...