AuroBlog - Aurous Healthcare Clinical Trials blog

MAY 7, 2024

When research tells us that our grandparents’ diets can affect our health decades later, it can be hard to shake the feeling that genetics spells out the trajectory of our wellbeing. But how much of our health is actually determined by our genes, and what effect does our lifestyle have, for better or worse? Can […]

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Genetics Gene Research Clinical Trials 207

Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

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AuroBlog - Aurous Healthcare Clinical Trials blog

JUNE 25, 2024

A woman in Colombia with rare genetic resistance to Alzheimer’s disease may have more company than scientists thought.

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Gene Genetics Scientist Research 209

Bio Pharma Dive

APRIL 5, 2023

The deal hands Ginkgo technology for discovering and engineering capsids — the outer shell that protects the helpful genetic material in gene therapies.

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Gene Therapy Gene Engineer Genetics 256

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

JULY 28, 2023

The British pharma is deepening its investment in genetic medicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back.

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Gene Therapy Gene Genetics Medicine 246

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 196

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

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 258

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 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 278

Pharmaceutical Technology

OCTOBER 19, 2022

Eli Lilly and Company has signed a definitive agreement for the acquisition of all outstanding shares of precision genetic medicine firm Akouos for a total deal value of up to nearly $610m or up to $15.50 Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss.

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Gene Therapy Gene Development Genetics 298

pharmaphorum

NOVEMBER 13, 2024

More than two years after its approval in Europe, PTC Therapeutics' gene therapy for rare genetic disorder AADC deficiency will soon be made available to patients in the US.The FDA has approved PTC's eladocagene exuparvovec – known as Upstaza in Europe – under the Kebilidi trade name in the US.

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Gene Therapy Gene Genetics 92

Bio Pharma Dive

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

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Gene Therapy Gene Genetics Trials 278

Pharma Mirror

APRIL 28, 2021

The post AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases appeared first on Pharma Mirror Magazine. and Europe. Catalent will further support process optimization and look to reduce material.

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Gene Therapy Genetic Disease Genetics Gene 130

Bio Pharma Dive

APRIL 25, 2024

“With each passing year, we're more committed to becoming a serious player in the genetic medicine space,” a Regeneron executive said.

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Gene Editing Gene Genetics Medicine 301

Pharmaceutical Technology

DECEMBER 23, 2022

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.

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Genetics Medicine Development Gene Therapy 328

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. Topic sponsors are not involved in the creation of editorial content.

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Gene Therapy Gene Development Manufacturing 256

AuroBlog - Aurous Healthcare Clinical Trials blog

DECEMBER 21, 2022

Scientists have found an extremely subtle twist in the genetics of aging cells, one that seems to make them increasingly less functional as time goes on.

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Genetics Gene Scientist Research 183

Bio Pharma Dive

JULY 21, 2021

The approval of Skysona for a genetic brain disease is a milestone for one of gene therapy's pioneering companies, but isn't expected to turn Bluebird's financial fortunes around.

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Pharmaceutical Technology

APRIL 15, 2024

Discover the groundbreaking gene therapy patent by Ginkgo BioWorks Inc for treating NPC1 using AAV vectors. Learn how this innovative approach offers hope for rare genetic disorders like Neimann-Pick Disease Type C.

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Gene Sequencing Gene Gene Therapy Genetics 130

Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Development Manufacturing 242

Pharmaceutical Technology

JANUARY 24, 2024

NAYA’s acquisition includes Florida Biotechnologies’ AAV gene therapy for the treatment of a rare genetic disorder.

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Gene Therapy Gene Genetics 130

BioSpace

MARCH 24, 2024

Ubiquitous potential, possible safety advantages and the recent growth of cell and gene therapy are driving investment in a different type of genetic editing.

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Gene Editing Gene Gene Therapy Genetics 140

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Gene Editing Gene Gene Therapy In-Vivo 189

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP. By Cytiva Thematic.

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Gene Editing Gene Gene Therapy Genetics 130

Fierce Pharma

MAY 8, 2024

After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D., received a simple email from Commissioner Robert Califf, M.D. | After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D.,

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Gene Therapy Gene Genetic Disease Genetics 130

Worldwide Clinical Trials

DECEMBER 12, 2022

Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. What are the benefits and limitations to using genetic testing? It is important to consider the type of testing performed (e.g.,

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Clinical Trials Clinical Trials Genetics Trials 130

pharmaphorum

MARCH 26, 2024

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of underlying genetic mutations.

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Gene Therapy Gene FDA Approval Genetics 128

Pharmaceutical Technology

JUNE 13, 2023

Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing. Amongst it was AGTC’s lead clinical candidate, AFTC-501, an adeno-associated virus (AAV) gene therapy for XLRP. Beacon Therapeutics is the third gene therapy company Syncona has launched.

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Gene Therapy Gene Trials Clinical Trials 246

Bio Pharma Dive

JANUARY 24, 2024

An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.

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Gene Therapy Gene Genetics Medicine 293

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 249

Bio Pharma Dive

DECEMBER 8, 2023

million, while a competing genetic medicine also cleared Friday is priced at $3.1 Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2

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Gene Therapy Gene Genetics Medicine 300

Medical Xpress

MARCH 27, 2023

Increasing use of genetic testing means people may discover they have a gene variant associated with some types of cardiovascular disease (CVD).

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Genetics Gene Genome Genomics 81

Pharmaceutical Technology

SEPTEMBER 30, 2022

After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.

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Gene Therapy FDA Approval Gene Genetic Disease 289

Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space.

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Gene Therapy Gene Medicine Genetics 245

Scienmag

SEPTEMBER 18, 2020

Two active genetics strategies help address concerns about gene-drive releases into the wild Credit: Ana Silva In the past decade, researchers have engineered an array of new tools that control the balance of genetic inheritance.

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Genetics Gene Engineer Research 86