Bio Pharma Dive

JUNE 11, 2025

The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to help overcome the packaging constraints of current genetic medicines.

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Gene Therapy Packaging Packaging Gene 236

Pharmaceutical Technology

MAY 28, 2025

3PL logistics need to invest in new technology and upskilling programs to scale their cell and gene therapy services.

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Gene Therapy Gene 130

Bio Pharma Dive

JULY 14, 2025

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather than a roadblock.”

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Gene Therapy Manufacturing Gene 152

Bio Pharma Dive

JULY 16, 2025

States representing about 84% of Medicaid beneficiaries are participating in an initiative that enables the U.S. government to negotiate pay-for-performance deals with the manufacturers of products like Casgevy and Lyfgenia.

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Gene Therapy Gene Manufacturing Production 170

Pharmaceutical Technology

JUNE 17, 2025

Supply chains for a new generation of therapies are complex, costly, and require meticulous management to ensure patient safety and regulatory compliance. AI can transform cell and gene therapy (CGT) orchestration by automating manual processes, improving productivity, enhancing supply chain visibility and improving scalability.

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Gene Therapy Compliance Gene Production 255

Medical Xpress

JUNE 23, 2025

Skin grafts genetically engineered from a patient's own cells can heal persistent wounds in people with an extremely painful dermatologic disease, a Stanford Medicine-led clinical trial has shown.

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Genetic Engineering Gene Therapy Dermatology Trials 123

Pharmaceutical Technology

JUNE 24, 2025

Gene therapy fill-finish processing presents a range of unique technical challenges due to the complexity and sensitivity of the drug substances involved.

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Gene Therapy Gene Production Drugs 130

pharmaphorum

JULY 16, 2025

Sanofi has been granted a speedy review from the FDA for a gene therapy for geographic atrophy, a major cause of blindness

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Gene Therapy Gene 51

Pharmaceutical Technology

MAY 16, 2025

The deal gives Lilly access to the companys RNA-editing platform to develop therapies for sensorineural hearing loss.

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Gene Therapy RNA Gene Development 147

Worldwide Clinical Trials

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. This can be a devastating outcome when trying again just isn’t possible.

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Gene Therapy Clinical Supply Trials Gene 182

pharmaphorum

JUNE 25, 2025

Sangamo plans to file for approval of its gene therapy for Fabry disease next year, but a launch could depend on finding a commercial partner.

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Gene Therapy Gene 97

XTalks

MAY 9, 2025

The FDA has granted approval to Abeona Therapeutics Zevaskyn (prademagene zamikeracel, also known as pz-cel), marking it as the first and only cell-based gene therapy approved for the rare skin disease recessive dystrophic epidermolysis bullosa (RDEB).

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Gene Therapy Gene FDA Approval Dermatology 90

STAT News

JULY 11, 2025

Its off-the-shelf cell therapy, called deramiocel, would have been the first treatment cleared specifically for the cardiomyopathy, a serious heart condition, that is associated with Duchenne. It could have also been used alongside other Duchenne drugs or gene therapies. 

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Gene Therapy Gene Marketing Drugs 118

The Pharma Data

JUNE 24, 2025

This alliance aims to address mounting challenges in the development and commercialization of mAb therapies and gene therapies, particularly those involving AAV vectors. However, the gene therapy sector faces its own unique set of obstacles.

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Gene Therapy Gene Development Manufacturing 40

STAT News

JUNE 11, 2025

In December 2023, a groundbreaking announcement grabbed headlines : After years of anticipation, the Food and Drug Administration approved the first gene therapies for treating sickle cell disease, offering hope of eliminating life-altering symptoms. Never mind the million-dollar gene therapy. Read the rest…

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Gene Therapy Gene Drugs 114

Pharmaceutical Technology

JUNE 12, 2025

The FDA has approved Capsida Biotherapeutics' investigational new drug (IND) application for CAP-003 to treat Parkinson’s disease (PD).

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Gene Therapy FDA Approval Gene Drugs 130

Scienmag

JULY 1, 2025

Such temporal and spatial regulation addresses one of the most significant challenges in gene therapy: minimizing off-target effects while achieving robust gene modulation in specific cell populations or tissues.

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Gene Editing Gene Gene Therapy Genomics 59

Fierce Pharma

DECEMBER 2, 2024

The FDA is investigating reports of patients develo | The FDA is investigating reports of patients developing blood cancer after receiving bluebird bio’s gene therapy Skysona and is evaluating the need for “further regulatory action,” the U.S. agency said last week.

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Gene Therapy Clinical Trials Clinical Trials Gene 126

Scienmag

JUNE 17, 2025

The landscape of gene therapy is undergoing a revolutionary transformation fueled by the convergence of synthetic biology, artificial intelligence, and biomedical engineering. Unlike traditional gene therapies that often rely on static expression systems, these circuits enable real-time, fine-tuned control of gene dosage.

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Genetics Gene Therapy Gene Expression Gene 59

XTalks

JANUARY 16, 2025

Meanwhile, Medtronics BrainSense Adaptive DBS, newly approved in Europe , could help advance neuromodulation therapy for Parkinsons with real-time brain stimulation adjustments as it becomes available in 2025.

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Trials Clinical Trials Clinical Trials Gene Therapy 104

BioTech 365

JUNE 30, 2025

Sensorion Announces Preliminary Positive Data from the First Cohort of the Audiogene Phase 1/2 Gene Therapy Clinical Trial Sensorion Announces Preliminary Positive Data from the First Cohort of the Audiogene Phase 1/2 Gene Therapy Clinical Trial SENS-501, including the surgical … Continue reading →

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Gene Therapy Clinical Trials Clinical Trials Gene 52

pharmaphorum

JULY 14, 2025

A UK consortium has been set up to tackle a major issue in the development of cell and gene therapies (CGT) – a lack of manufacturing capacity.

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Manufacturing Gene Therapy Gene Development 87

Bio Pharma Dive

MAY 30, 2025

Elsewhere, Stealth Biotherapeutics and Taysha Gene Therapies each think they've found a path to FDA approval. Keros will also stop work on a cardiovascular disease drug.

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Gene Therapy FDA Approval Gene Drugs 152

Bio Pharma Dive

JUNE 23, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Sign up A valid email address is required. Please select at least one newsletter.

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Gene Therapy Drugs Gene Life Science 130

FDA Law

DECEMBER 15, 2024

Valentine We recently published the first part of our review of FDAs draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. If you make a tissue product you may produce one lot per patient where a gene therapy may only need a handful of lots through Phase 3.

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Gene Therapy Gene Production Manufacturing 52

XTalks

JANUARY 6, 2025

The diversity of CF treatments is expanding with investigational therapies such as Spirovants inhaled gene therapy SP-101 in a Phase I/II trial and Kither Biotechs inhaled peptide therapy KIT2014 in a Phase I trial.

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Gene Therapy Protein Genetic Disease Gene 105

XTalks

APRIL 15, 2025

In early 2025, Pfizer discontinued its AAV gene therapy Beqvez (fidanacogene elaparvovec) for hemophilia B, citing limited uptake among physicians and patients. Setbacks and Strategic Pivots Progress hasnt come without setbacks.

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Gene Therapy Gene Clinical Trials Clinical Trials 66

XTalks

NOVEMBER 29, 2024

Roche’s acquisition of Poseida Therapeutics comes amid a wave of high-profile investments in cell and gene therapy. This CD19-directed therapy generates CAR-T cells directly within the patient’s body, addressing the scalability challenges of traditional ex vivo approaches. Meanwhile, Novartis acquired Kate Therapeutics in a $1.1

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Gene Therapy Gene Gene Editing Genetic Engineering 100

Bio Pharma Dive

JULY 9, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime.

Drugs 155

Drugs Protein Gene Therapy Clinical Development 155

STAT News

JUNE 20, 2025

The ouster of the Food and Drug Administration’s chief regulator of cell and gene therapies earlier this week came immediately after a disagreement with her boss over the review of a cell therapy for Duchenne muscular dystrophy, STAT has learned. 

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Gene Therapy Regulation Gene Development 126

Scienmag

JULY 7, 2025

The implications extend beyond traditional gene therapy into synthetic biology and functional genomics. The ability to selectively manipulate individual nucleotides within native chromatin landscapes catalyzes new experimental designs investigating gene regulation, epistatic interactions, and evolutionary dynamics.

Genomics 59

Genomics Genome Genotype Engineer 59

XTalks

APRIL 8, 2025

Specialized facilities such as gene therapy dosing centers or advanced MRI sites can serve as treatment hubs, even if they are far from patients homes. In gene therapy studies, for instance, patients may need to travel to specialized dosing locations outside their home country.

Clinical Trials 88

Clinical Trials Clinical Trials Trials Gene Therapy 88

Scienmag

JUNE 19, 2025

Subject of Research : In vivo generation of CAR T cells using targeted lipid nanoparticles for cancer and autoimmune disease treatment Article Title : In vivo CAR T cell generation to treat cancer and autoimmune disease News Publication Date : 19-Jun-2025 Web References : [link] Keywords : CAR T cells, in vivo engineering, lipid nanoparticles, mRNA (..)

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In-Vivo Engineer Manufacturing RNA 52

Pharma Times

JULY 8, 2025

Tags Related posts Clinical Researcher of the Year – The Americas Jul 8, 2025 First patient dosed in trial for novel dry AMD treatment PST-611 Jul 7, 2025 Eladynos recommended for osteoporosis treatment in Scottish NHS Jul 7, 2025 First German haemophilia B patient receives gene therapy HEMGENIX Jul 3, 2025 News alerts This field is required.

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Trials Gene Therapy Gene Clinical Research 79

Pharma Times

JUNE 19, 2025

The collaboration agreement will see eXmoor, an integrated cell and gene therapy contract development and manufacturing organisation, support Signadori in the transition from discovery through to good manufacturing practice (GMP)-compliant manufacturing of its lead candidate.

Gene Therapy 119

Gene Therapy FDA Approval Gene Manufacturing 119

STAT News

JUNE 20, 2025

Want to stay on top of the science and politics driving biotech today?    Sign up  to get our biotech newsletter in your inbox. Hello from the Psychedelic Science Conference in Denver! A fascinating ideological clash underway here, if I do say so myself — counterculture meets corporate science. 

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Gene Therapy Gene Drugs 60

Scienmag

JUNE 21, 2025

Furthermore, gene therapy approaches aimed at correcting dysfunctional gene expression profiles in interneurons could emerge as viable next-generation treatments. In addition to therapeutic insights, this study holds promise for improving diagnostic paradigms.

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Gene Expression Gene Regulation Research 80