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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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

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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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.

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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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

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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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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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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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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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. The treatment is being researched by scientists collaborating from American Gene Technologies, the Laboratory of Immunoregulation and the National Institute of Allergy and Infectious Diseases.

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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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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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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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XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

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Pharmaceutical Technology

APRIL 5, 2023

But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. AZ: Cell and gene therapies often come with a high price.

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Camargo

JULY 30, 2021

In the Pediatric Study Plan, you have a defined timeline and a defined plan, a clinical development plan that has to be approved by the FDA. That has to do a lot with the work in gene therapy and rare disease and the ultra-rare space because there are 7,000 rare diseases that have been identified.

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Pharmaceutical Technology

APRIL 5, 2023

Caribou Biosciences chief medical officer Syed Rizvi said: “Fast Track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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