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. 2026: 15 staffers.

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Gene Therapy Gene Development Production 184

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.

Clinical Development 132

Clinical Development Gene Therapy Development Gene 132

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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Gene Therapy Gene Clinical Development Marketing 130

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 173

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., Deyle, M.D.,

Gene Therapy 130

Gene Therapy Gene Genetics Clinical Development 130

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.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

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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Gene Therapy Gene Packaging Packaging 155

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

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.

Gene Therapy 100

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.

Gene Therapy 100

Gene Therapy Gene Clinical Development Development 100

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.

Gene Therapy 98

Gene Therapy Gene Clinical Development Development 98

Pharmaceutical Technology

DECEMBER 30, 2024

ICG-124 is under clinical development by iCell Gene Therapeutics and currently in Phase I for T-Cell Acute Lymphocytic Leukemia (T-Cell Acute Lymphoblastic Leukaemia).

Gene 100

Gene Clinical Development Development 100

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.

Clinical Development 110

Clinical Development Development Clinical Trials Clinical Trials 110

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Editing Gene Gene Therapy In-Vivo 130

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. Clinical holds are becoming more common, especially in gene therapy programs.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

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. The one-shot therapy aims to treat GA by delivering the gene for complement factor I (CFI) to the eye using an adeno-associated virus (AAV) vector.

Gene Therapy 105

Gene Therapy Gene Licensing Licensing 105

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

Pharmaceutical Technology

SEPTEMBER 14, 2023

BCMA-GPRC5D CAR-T Cells is under clinical development by Guangzhou Bio-gene Technology and currently in Phase II for Refractory Multiple Myeloma.

Gene 100

Gene Clinical Development Development 100

Pharmaceutical Technology

SEPTEMBER 14, 2023

BCMA-GPRC5D CAR-T Cells is under clinical development by Guangzhou Bio-gene Technology and currently in Phase II for Relapsed Multiple Myeloma.

Gene 100

Gene Clinical Development Development 100

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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Development Gene Therapy Protein Gene 246

Pharmaceutical Technology

MARCH 10, 2023

Zavzpret is claimed to be the first and only calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray approved to treat migraine. It is a third-generation, selective and structurally unique, small molecule CGRP receptor antagonist and is currently under clinical development with oral and intranasal formulations.

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FDA Approval Pharmacy Trials Clinical Development 246

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. While cell and gene therapies are currently much more expensive than traditional medical treatments, the ultimate savings could be significant.

Gene Therapy 130

Gene Therapy Gene Medicine Manufacturing 130

BioPharma Reporter

NOVEMBER 16, 2021

have received the green light from Health Canada for their Clinical Trial Application for VCTX210: an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D). CRISPR Therapeutics and ViaCyte, Inc.

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Gene Editing Gene Clinical Trials Clinical Trials 111

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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In-Vivo Gene Therapy Gene RNA 246

Pharmaceutical Technology

NOVEMBER 28, 2022

According to the deal, C4XD is entitled to get up to $16m in pre-clinical milestone payments before the commencement of the first clinical trial. . on meeting clinical development and commercial milestones in the future, in addition to tiered royalty payments, upon the marketing of the product.

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Development Gene Expression Licensing Licensing 264

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.

Gene Therapy 98

Gene Therapy Manufacturing Gene Clinical Development 98

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.

Gene Therapy 246

Gene Therapy Gene Clinical Development Development 246

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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Gene Therapy Clinical Development Gene Development 40

Pharmaceutical Technology

JULY 26, 2022

NPC is caused by mutations in the NPC1 gene (NPC type 1C) or the NPC2 gene (NPC type 2C) and is inherited in an autosomal recessive manner. Most cases are detected during childhood and progress to cause life-threatening complications by the second or third decade of life.

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Marketing Gene Branding Clinical Development 306

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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Manufacturing Gene Therapy Clinical Development Gene 130

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. This gap calls for well-planned logistical solutions.

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Clinical Trials Clinical Trials Trials Gene Therapy 88

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. For a full look at the cell and gene therapy landscape, check out our webinar titled: “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapy.”). (For Check it out!

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Gene Therapy Gene Development Research 130

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

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

Gene Therapy 40

Gene Therapy Gene Clinical Development Development 40

Outsourcing Pharma

AUGUST 11, 2020

American Gene Technologies has received the agencyâs approval to launch the first human trial for its lead HIV program, starting in Baltimore and DC.

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Trials Gene Clinical Development Development 145

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.

Gene Therapy 130

Gene Therapy Gene In-Vivo Life Science 130

Pharmaceutical Technology

MAY 9, 2023

FibroGen has signed an exclusive licence agreement with Fortis Therapeutics for the FOR46 antibody-drug conjugate (ADC) that targets a new epitope on CD46, a protein-coding gene. FOR46 is a natural fit with our research and development capabilities and expertise.

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Drugs Clinical Development Antibody Protein 130