Bio Pharma Dive

MARCH 30, 2022

The biotech company plans to reduce its workforce by 30%, or by about 65 employees, in the latest example of a gene therapy developer restructuring.

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Gene Therapy Gene Research Development 279

Pharmaceutical Technology

NOVEMBER 8, 2022

Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Additionally, Neurophth will oversee the clinical trials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen.

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Gene Therapy Gene Development Genetics 305

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.

Gene Therapy 315

Gene Therapy Gene Trials Research 315

Bio Pharma Dive

JANUARY 21, 2021

Katherine High and Jude Samulski, two well-known gene therapy researchers, were longtime collaborators and competitors. Now they're colleagues at one of the world's largest drugmakers.

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Gene Therapy Gene Research 338

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

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

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

Bio Pharma Dive

APRIL 28, 2021

Far along in testing for a hemophilia treatment, BioMarin is expanding its gene therapy research into diseases of the central nervous system and aims to use some of the institute's technology.

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Gene Therapy Gene Development Research 315

Bio Pharma Dive

FEBRUARY 23, 2023

Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.

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Gene Therapy Gene Development Genetics 256

Bio Pharma Dive

JULY 17, 2023

Scribe will receive $40 million upfront from Sanofi in a collaboration initially focused on developing an in vivo gene editing treatment for sickle cell disease.

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Gene Editing Gene In-Vivo Research 237

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

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

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

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

Pharmaceutical Technology

MAY 16, 2023

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.

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Gene Therapy Gene Research Clinical Trials 227

Bio Pharma Dive

JANUARY 7, 2025

The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle cell and beta thalassemia.

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Gene Editing Gene Research Medicine 288

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 22, 2024

(Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows. Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth.

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Scientist Gene Genetics Research 244

AuroBlog - Aurous Healthcare Clinical Trials blog

JUNE 25, 2024

Family members with just a single copy of her version of a particular gene – what’s known as the Christchurch variant of apolipoprotein E (APOE) – seem to also be protected against genetically determined early-onset dementia, researchers […]

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Gene Genetics Scientist Research 245

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

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

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

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

AuroBlog - Aurous Healthcare Clinical Trials blog

APRIL 29, 2024

A unique genetic mutation in two siblings – that has never been seen in anyone else – has been discovered by UK researchers at the University of Exeter, pointing the way towards new treatment options for type 1 diabetes. The mutation is in the gene for a protein called programmed death-ligand 1 (PD-L1), and a […]

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Gene Genetics Protein Research 243

Bio Pharma Dive

JANUARY 27, 2025

Taking control of O2 — ushering in the future of cell therapy research.

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Gene Therapy Research Gene 245

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 360

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

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 Gene Therapy Research 246

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

Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

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AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 9, 2024

The convergence of gene therapies and clinical research is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.

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Gene Therapy Gene Genetics Clinical Research 244

Bio Pharma Dive

OCTOBER 28, 2021

The two institutions will work with drugmakers and non-profit groups on a wide-ranging initiative meant to make the complex treatments easier and less costly produce.

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Gene Therapy Gene Research 294

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 245

Pharmaceutical Technology

JULY 31, 2024

Gene therapy research is expensive. Biotechs invest millions of dollars in early research to develop therapies for conditions that might be rare. But when funds are limited and the market potential is dim, such programs are often abandoned by companies, leaving patients and investigators in a bind.

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Gene Therapy Research Gene Marketing 245

Bio Pharma Dive

FEBRUARY 25, 2021

Sean Nolan and Joe Nolan, former leaders at the Zolgensma developer, are behind Jaguar Gene Therapy, which plans to research treatments for a rare metabolic disease as well as genetically linked autism and diabetes.

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Gene Therapy Gene Genetics Development 242

Bio Pharma Dive

AUGUST 1, 2024

Wilson, who founded UPenn’s gene therapy program three decades ago, will step down to start two new spinouts, Gemma Bio and Franklin Biolabs.

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Gene Therapy Gene Research 177

Bio Pharma Dive

APRIL 26, 2022

Apertura Gene Therapy, a new company formed by Deerfield, is hoping a suite of next-generation tools will help it stand out at a time when this area of research has fallen out of favor with some public investors.

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Gene Therapy Gene Research 246

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