Pharmaceutical Technology

JUNE 13, 2023

The US Food and Drug Administration (FDA) approved Ferring Pharmaceuticals’ Prior-Approval Supplement (PAS) to the Biologics License Application (BLA) for the bladder cancer gene-therapy Adstiladrin (nadofaragene firadenovec). The therapy received approval based on positive Phase III trial (NCT02773849) results.

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Camargo

DECEMBER 20, 2021

Many of the advancements included in PDUFA VII are driven in large part by an increase in the development of innovative products such as cell and gene therapies. The increase is designed to strengthen CBER’s capacity and capabilities for regulating cell and gene therapies. 2024: 48 staffers. 2025: 29 staffers.

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BioSpace

JULY 22, 2021

Shares of Adverum Biotechnologies plunged more than 20% in premarket trading after the company announced it was revising its clinical development plan for investigational gene therapy candidate ADVM-022 based on safety concerns in patients with diabetic macular edema (DME).

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Pharma Mirror

JANUARY 30, 2022

Ben Beckley, Global Lead at EmerGENE explores the market challenges holding back Cell and Gene Therapy (C&GT) from taking its place as an established treatment area. Some 3,366 treatments are currently in preclinical and clinical development[1]. As a treatment area, C&GT has shown robust growth in recent years.

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Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

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Pharma Mirror

OCTOBER 26, 2021

a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options, has formed a new research collaboration with Mayo Clinic. Castle Creek Biosciences Inc.,

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Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Around 40% of clinical holds are for gene therapy programs.

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Gene Therapy Gene Clinical Trials Clinical Trials 130

Pharmaceutical Technology

MAY 17, 2024

Gene Therapy for Parkinson’s Disease is under clinical development by Innervate Therapeutics and currently in Phase II for Parkinson's Disease.

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Gene Therapy Gene Clinical Development Development 100

Bio Pharma Dive

AUGUST 6, 2024

The company plans to limit sales of the hemophilia gene therapy to the U.S., Italy and Germany, while ending most clinical development work.

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Sales Gene Therapy Clinical Development Gene 169

BioPharma Reporter

JUNE 20, 2022

The Cell and Gene Therapy Catapult (CGT Catapult) and the UK Dementia Research Institute (UK DRI) have announced a collaboration to accelerate clinical development of adeno-associated virus (AAV) based gene therapies for dementia.

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Gene Therapy Gene Clinical Development Development 98

Pharmaceutical Technology

DECEMBER 26, 2024

Gene Therapy to Target GD2 for Oncology is under clinical development by Bristol-Myers Squibb and currently in Phase I for Sarcomas.

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Gene Therapy Gene Clinical Development Development 100

Pharmaceutical Technology

DECEMBER 26, 2024

Gene Therapy to Target GD2 for Oncology is under clinical development by Bristol-Myers Squibb and currently in Phase I for Osteosarcoma.

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Gene Therapy Gene Clinical Development Development 100

pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

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Gene Therapy Gene Manufacturing Clinical Trials 97

pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development.

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Gene Therapy Gene DNA Development 105

pharmaphorum

DECEMBER 22, 2021

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. Last year, it also signed a licensing deal with Dyno Therapeutics for its AAV capsid-based gene delivery technology focused on ocular diseases.

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Gene Therapy Gene Licensing Licensing 105

Pharmaceutical Technology

AUGUST 18, 2022

A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis. The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study.

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Gene Therapy Gene Genotype In-Vivo 246

pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one of three UK sites for the clinical trial and part of 55 globally, across 15 countries.

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Gene Therapy Gene Trials Protein 102

Medical Xpress

APRIL 26, 2023

A successful gene therapy trialed at Michigan State University in dogs with an inherited eye disease is ready to be developed for clinical use in human patients with a rare condition called retinitis pigmentosa.

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Clinical Development Gene Therapy Development Gene 133

Pharmaceutical Technology

JANUARY 26, 2023

Cell and gene therapies are predicted to be the future of medical treatment by providing the body with the means to repair itself and recover from a range of serious conditions and severe diseases. Often, cell and gene therapies are spoken of as miracle treatments, yet this progress has hardly occurred overnight.

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Gene Therapy Gene Medicine Manufacturing 130

BioPharma Reporter

NOVEMBER 7, 2022

Charles River Laboratoriesâ growing gene therapy manufacturing capabilities have landed it a deal with the eye disease biotech Nanoscope Therapeutics.

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Gene Therapy Manufacturing Gene Clinical Development 98

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

MAY 19, 2023

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

APRIL 17, 2023

We believe having access to the public markets will help enable us to fund our clinical development in advanced neurodegenerative diseases and support the launch of our off-the-shelf allogenic programme in oncology.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Clinical Development Development 246

XTalks

JUNE 4, 2024

Oren Cohen, MD, FIDSA CMO & President of Clinical Pharmacology Fortrea Fortrea is a provider of comprehensive Phase I through IV clinical trial management, clinical pharmacology, market access solutions and other enabling services. Our mission at Fortrea is to deal with and manage the complexity in the space,” says Dr.

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Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

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Worldwide Clinical Trials

MAY 20, 2022

The past few years we’ve had exciting opportunities to work on more cell and gene therapy programs for our sponsors. The industry is just at the tip of the iceberg for exploring the potential of these therapies in revolutionizing how we treat disease, and as the approvals start to trickle in, the pipeline only grows.

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BioPharma Reporter

JULY 9, 2024

The fundraising will accelerate the clinical development of Beacon Therapeuticsâ lead gene therapy candidate, which is currently in phase 2/3 trials.

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Gene Therapy Gene Clinical Development Trials 52

pharmaphorum

JANUARY 11, 2021

While bluebird has conducted pioneering work in gene therapy for blood disorders and in cancer cell therapy, products have been delayed by issues with filing data for the FDA. The post bluebird bio to split into oncology and gene therapy specialists appeared first on. million price tag. million price tag.

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Gene Therapy Gene Genetic Disease Manufacturing 66

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.

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Clinical Trials Clinical Trials Trials Gene Therapy 88

pharmaphorum

NOVEMBER 12, 2020

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its gene therapy drive with Handl acquisition appeared first on.

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BioTech 365

DECEMBER 16, 2021

Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-118 for the Treatment of CLN1 Disease Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-118 for the Treatment of CLN1 Disease CTA approved by Health Canada in November 2021; … Continue reading →

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pharmaphorum

JUNE 27, 2022

The safety of Astellas’ gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845. The post Another Astellas gene therapy hits a safety hurdle appeared first on.

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Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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pharmaphorum

FEBRUARY 8, 2022

With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas has said that an interim readout in a phase 1/2 trial of its Pompe disease candidate AT845 hasn’t found any cause for concern. The post Astellas says Pompe gene therapy clears safety hurdle appeared first on.

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Gene Therapy Gene Immune Response Licensing 52

Pharma Mirror

JULY 23, 2021

The initial phase of the expansion will see the installation of two 2,000-liter single-use bioreactors within new, purpose-built manufacturing suites and associated investments to support early-phase clinical development as well as late-stage and commercial tech transfers. SOMERSET, N.J. It will also.

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XTalks

JANUARY 3, 2024

Novartis and Voyager Therapeutics have struck a licensing deal that will see Novartis pay Voyager $100 million upfront to develop gene therapy candidates targeting Huntington’s disease (HD) and spinal muscular atrophy (SMA). billion with preclinical, development, regulatory and sales milestones. Sandrock, Jr.,

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Gene Therapy Gene Licensing Licensing 52

BioTech 365

JANUARY 27, 2022

Taysha Gene Therapies Announces Positive Initial Biomarker Data For TSHA-101, the First Bicistronic Gene Therapy in Clinical Development, Demonstrating Normalization of ?-Hexosaminidase

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