Bio Pharma Dive

NOVEMBER 3, 2021

Data readouts for Pfizer's hemophilia gene therapies are now expected later than previously forecast, while the company also works through modifying a trial of its Duchenne treatment.

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Gene Therapy Gene Trials Research 305

Bio Pharma Dive

MAY 7, 2024

The company is working with trial researchers to investigate further. Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly.

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Gene Therapy Gene Trials Research 355

Bio Pharma Dive

MARCH 6, 2023

The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.

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Gene Therapy Gene Licensing Licensing 263

Bio Pharma Dive

JANUARY 13, 2022

As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines.

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Gene Therapy Gene Research 351

Pharmaceutical Technology

APRIL 2, 2024

In this issue: High-profile gene therapies that use AAV vectors, new research into HIV-TB coinfection treatments, traceability technologies for the supply chain, and more.

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Gene Therapy Gene Research 130

Bio Pharma Dive

MAY 17, 2023

Spun out of the lab of former UCSF researcher Nicole Paulk, the biotech is targeting brain and eye tumors with a new kind of treatment it has dubbed “immuno-gene therapy.”

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Gene Therapy Gene Research 295

Bio Pharma Dive

JULY 28, 2023

The company will lay off 87% of its workforce and halt further research, including for a gene editing therapy to treat the rare disease PKU.

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Gene Editing Gene Sales Research 246

Bio Pharma Dive

MARCH 24, 2022

At least nine biotechs working in cell or gene therapy have announced layoffs, cost cuts or restructured their research since December — restructurings that have coincided with a stock market downturn.

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Gene Therapy Gene Marketing Research 361

Bio Pharma Dive

JULY 19, 2023

The agency veteran, who led the Office of Tissues and Advanced Therapies during a boom in gene and cell therapy research, retired in March.

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Gene Therapy Gene Research 246

Pharmaceutical Technology

MAY 4, 2023

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.

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Gene Therapy Gene Development Manufacturing 256

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. These insights were shared through several sessions, in presentation tracks specifically dedicated to “Community and Capacity Building” and “Becoming a Research Ready Organization.”

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Gene Gene Editing Gene Therapy Clinical Trials 130

Bio Pharma Dive

DECEMBER 8, 2021

Passage Bio and the University of Pennsylvania's Gene Therapy Program said they have begun preclinical research aimed at simultaneously developing multiple approaches to treating Huntington's.

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Gene Therapy Gene Development Research 343

Bio Pharma Dive

SEPTEMBER 3, 2021

A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field.

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Gene Therapy Gene Regulation Research 362

Pharmaceutical Technology

OCTOBER 19, 2022

Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss. It has combined expertise in otology, gene therapy and inner ear drug delivery to meet the requirements of individuals with disabling hearing loss globally.

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Gene Therapy Gene Development Genetics 298

Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Development Genetics 264

Bio Pharma Dive

JULY 20, 2022

The deal, which is worth $60 million upfront, broadens Vertex’s reach into gene editing and expands Verve’s research beyond heart disease.

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Gene Editing Gene Development Drugs 323

Bio Pharma Dive

APRIL 1, 2022

Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.

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Gene Therapy Gene Development Genetics 272

Bio Pharma Dive

APRIL 3, 2023

Cell & gene therapy clinical research may help treat diseases and change how we approach healthcare.

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Gene Therapy Gene Clinical Trials Clinical Trials 256

Bio Pharma Dive

APRIL 4, 2022

Safety issues in clinical trials cast doubt on the ability of gene therapy to safely mitigate disease, prompting further research into understanding the virus-host interaction that resulted in new generations of viral vectors.

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Gene Therapy Gene Clinical Trials Clinical Trials 246

Pharmaceutical Technology

JUNE 19, 2023

Indian pharmaceutical company Laurus Labs has signed a memorandum of agreement (MoA) with the Indian Institute of Technology, Kanpur (IIT Kanpur) to bring new gene therapy products to the market. Laurus Labs CEO Satyanarayana Chava stated: “This collaboration exhibits our commitment towards the cell and gene therapy (CGT) space.

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Gene Therapy Gene Production Pharma Companies 246

Pharmaceutical Technology

FEBRUARY 1, 2024

The French government grant will cover preclinical and clinical research for Vivet’s gene therapy for cerebrotendinous xanthomatosis.

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Gene Therapy Gene Development Clinical Research 147

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors. Sanofi is one of the leading patent filers working with viral vectors.

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Gene Therapy Gene Genome Genomics 246

Bio Pharma Dive

JUNE 26, 2021

Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective, a culmination of years of scientific research.

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Gene Editing Gene Medicine Research 363

Pharmaceutical Technology

FEBRUARY 2, 2023

If things go as per plan, in a few months, the US Food and Drug Administration (FDA) will deliberate on the first-of-its-kind CRISPR-based gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The post Magazine: CRISPR gene therapies cut through in 2023 appeared first on Pharmaceutical Technology.

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Gene Therapy Gene Pharma Companies Clinical Trials 246

Pharmaceutical Technology

MAY 30, 2023

Amplo Biotechnology has received a fast track phase I/II STTR grant from the NIH-NIAMS [National Institutes of Health’s National Institute of Arthritis and Musculoskeletal and Skin Diseases] for its gene therapy AMP-201. The company will receive substantial funding to advance AAV-ColQ gene therapy.

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Gene Therapy Gene Development Clinical Trials 244

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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Gene Therapy Gene Gene Editing Manufacturing 243

STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham. Continue to STAT+ to read the full story…

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Gene Editing Gene Genetic Disease Gene Therapy 126

Pharmaceutical Technology

MARCH 10, 2023

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. In the past, researchers have often had to seek help from outside the UK, thus delaying clinical trials and patient access.

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Gene Therapy Manufacturing Gene Clinical Trials 264

Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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Gene Therapy Gene Development Engineer 237

Bio Pharma Dive

JULY 12, 2023

The decision to explore “strategic alternatives” comes about six weeks after the company sold its most advanced treatment to Novartis for nearly $90 million.

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Gene Therapy Sales Gene Research 246

Bio Pharma Dive

JUNE 30, 2021

The companies aim to develop therapies for complement-driven diseases, a specialty of Apellis and relatively new research ground for gene editing.

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Gene Editing Gene Development Research 300

Medical Xpress

NOVEMBER 16, 2022

Researchers at the National Institutes of Health have successfully identified differences in gene activity in the brains of people with attention deficit hyperactivity disorder (ADHD).

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Gene Research Genome Genomics 98

Bio Pharma Dive

NOVEMBER 5, 2020

Sarepta and Pfizer are advancing their experimental treatments into late-stage trials, while others are accelerating research in what's become one of gene therapy's most active fields.

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Gene Therapy Gene Trials Research 315

Medical Xpress

APRIL 11, 2023

University of Virginia School of Medicine researchers have identified a gene that plays a crucial role in determining the risk for heart attacks, deadly aneurysms, coronary artery disease and other dangerous vascular conditions.

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Gene Research Medicine Genetics 93

AuroBlog - Aurous Healthcare Clinical Trials blog

FEBRUARY 14, 2023

Age catches up with us all eventually, but in some people the right genes can make that chase into our twilight years a relatively leisurely one. A few years ago Italian researchers discovered something special about people who live well into their 90s and beyond: they commonly have a version of a gene called BPIFB4 […]

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Gene Research Clinical Trials Clinical Trials 194

Bio Pharma Dive

MARCH 4, 2021

The planned factory is a strong sign of Biogen's interest in gene therapy, an area of research it has invested in through dealmaking over the past few years.

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Gene Therapy Gene Research 311