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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World of DTC Marketing

DECEMBER 29, 2020

OBSERVATION: Biologics can take a long time to develop but COVID vaccines have been in development for almost 50 years and novel approaches were used to develop these vaccines. Vaccines typically take 10 to 15 years to develop, test and release to the public. The coronavirus vaccines, however, took less than a year.

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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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Genetics Trials Clinical Trials Clinical Trials 189

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). This would allow the companies to boost the development of new therapy options for Rett syndrome and GAN patients.

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Gene Therapy Gene Development Genetics 264

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

AuroBlog - Aurous Healthcare Clinical Trials blog

FEBRUARY 18, 2024

Indian healthcare industry is of the view that emergence of genetic diagnostics will be a paradigm shift towards personalized medicine. Through the ever-evolving landscape of healthcare, the role of genetic diagnostics offers personalized insights and proactive solutions for disease management and prevention.

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Genetics Medicine Development Drugs 189

XTalks

DECEMBER 18, 2024

CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. While manageable, the condition requires lifelong treatment and monitoring to prevent complications and ensure healthy development.

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Genetics Hormones Trials Drugs 116

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 30, 2024

Researchers see a greater need for new generation therapeutics to propel precision medicine. The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics.

Medicine 189

Medicine Research Gene Therapy Genetics 189

Bio Pharma Dive

DECEMBER 12, 2021

But despite continued setbacks, drug developers are hopeful that genetic technologies and a better understanding of biology will bring new options in the not-too-distant future The fatal nerve disease has few treatments.

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Genetics Drugs Research Development 264

Pharmaceutical Technology

MARCH 24, 2023

Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.

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Genetics Medicine Development In-Vivo 130

pharmaphorum

AUGUST 6, 2024

Discover how Step Pharma is harnessing genetics and developing therapeutics targeting CTPS1 to advance cancer research and enhance clinical success. Learn more about their innovative approach.

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Genetics Research Development 119

Worldwide Clinical Trials

APRIL 8, 2025

In a recent webinar, Challenges of Drug Development in Early Parkinsons Disease , Tom Babic, MD, PhD , our Vice President of Scientific Solutions in Neuroscience, uses his 40+ years of experience to share invaluable insights into the current state and future directions of early Parkinsons disease (PD) research.

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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. This will improve accessibility to services for AAV-mediated gene therapy programmes.

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Gene Therapy Gene Development Manufacturing 256

Pharmaceutical Technology

FEBRUARY 9, 2023

ReviR Therapeutics has signed a research collaboration and option-to-license agreement with Asieris Pharmaceuticals to discover new oncology therapeutics. Its initial focus is to develop therapeutics in oncology, CNS, and genetically defined disease indications.

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Pharmaceutical Technology

FEBRUARY 20, 2023

While there is no cure to this neurodegenerative condition, academics and companies are pushing through with research that could help patients and their families. 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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Medical Xpress

DECEMBER 13, 2024

An international team of researchers has identified a key genetic mechanism that regulates the formation and migration of cranial neural crest cells, which are essential for developing facial structures.

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Genetics Development Regulation Research 104

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. The post Capsida Biotherapeutics and Prevail to develop CNS gene therapies appeared first on Pharmaceutical Technology.

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Gene Therapy Gene Development Engineer 237

XTalks

DECEMBER 19, 2024

Especially as we exited the blockbuster drug period of psychiatric development and trials were failing with huge placebo effects, I think we started realizing that the patients going into trials were not the real-world patients that were getting treated with drugs in the clinic.

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Clinical Trials Clinical Trials Trials Research 97

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 260

Medical Xpress

MAY 16, 2023

However, how the brain has developed over the course of evolution and which genes are responsible for the high cognitive abilities is still largely unclear. The better our understanding of the role of genes in brain development, the more likely it will be that we will be able to develop treatments for serious brain diseases.

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Development Genetics Gene Research 118

XTalks

MAY 23, 2024

One of the more intriguing developments in cancer research in recent years is the growing understanding of clonal hematopoiesis, a phenomenon where blood cells expand from a single clone due to genetic mutations. Clonal hematopoiesis increases in prevalence with age and can precede hematologic malignancies.

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Genetics Research Genome Genomics 114

Pharmaceutical Technology

MARCH 1, 2024

1910 Genetics has entered an agreement with Microsoft, aiming to revitalise pharmaceutical research and development (R&D) productivity.

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Genetics Production Development Research 130

Medical Xpress

MARCH 1, 2025

A team of researchers at the University Medical Center Schleswig-Holstein (UKSH), Kiel Campus, the Kiel University, and the Max Planck Institute for Molecular Genetics, Berlin, have developed an innovative method for real-time molecular genetic classification of brain tumors during surgery.

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DNA Genetics Development Research 90

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?

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Clinical Trials Clinical Trials Genetics Trials 130

Medical Xpress

DECEMBER 20, 2022

New research findings have revealed an underlying genetic cause for why some children who have had COVID-19 infection develop Multisystem Inflammatory Syndrome in Children (MIS-C), a rare but potentially life-threatening disease.

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Genetics Research Development 98

Pharmaceutical Technology

NOVEMBER 29, 2022

Researchers at City of Hope in the US have developed a new potential therapy to treat glioblastoma multiforme (GBM), a kind of brain cancer. These viruses are engineered genetically for killing cancer. The post City of Hope researchers develop new brain cancer therapy appeared first on Pharmaceutical Technology.

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Development Research Antibody Engineer 130

Worldwide Clinical Trials

JANUARY 6, 2025

Tom Zhang, PhD, Chief Scientific Officer, Large Molecule Bioanalysis Revolutionizing Therapeutic Development with Cutting-Edge Bioanalysis Automation Cutting-edge advancements in bioanalysis (BioA) and drug R&D are reshaping the future of preventative and interventional care, but how do we harness these to their fullest potential?

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Clinical Trials Clinical Trials Trials Genetics 130

Worldwide Clinical Trials

FEBRUARY 6, 2023

When using genetic screening to identify clinical trial volunteers, a sponsor’s obligations for further testing and disclosure of results to patients and families are unclear, especially when the results have no impact on medical management. Guidance in this area is much needed. Read the full article! Read the full article!

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Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics.

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Life Science Development Gene Therapy Genetics 162

Pharmaceutical Technology

MAY 17, 2023

Under the deal, Prevail obtains exclusive rights to use Scribe’s CRISPR X-Editing (XE) technologies to develop the medicines. The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets.

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Genetics Medicine In-Vivo Gene Therapy 130

Pharmaceutical Technology

OCTOBER 7, 2022

Surrozen and Boehringer Ingelheim have entered a partnership and licence agreement for researching and developing SZN-413 to treat retinal diseases. on meeting development, regulatory and commercial milestones, apart from sales-based royalty payments. Boehringer will make an upfront payment of $12.5m to Surrozen in return.

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Development Genetics Sales Antibody 147

Scienmag

JULY 1, 2022

Curing debilitating genetic diseases is one of the great challenges of modern medicine. During the past decade, development of CRISPR technologies and advancements in genetics research brought new hope for patients and their families, although the safety of these new methods is still of significant concern.

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Genetics Genetic Disease Medicine Development 97

FDA Law Blog

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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Production Clinical Development Development Regulation 69

pharmaphorum

JANUARY 24, 2023

Drug development has long been an issue for the pharma industry, due to the expense and the high failure rate of potential treatments. Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates.

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Genetics Drugs Genome Project Biobanking 98

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The option licensing agreement is based on early-stage clinical results.

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Development Genetic Disease Genetics Gene Therapy 130

Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

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Life Science Genetic Disease Genome Genomics 130

Pharmaceutical Technology

OCTOBER 18, 2022

Currently, the majority of PD therapeutics and biomarkers are developed to diagnose and treat PD patients that show Lewy body pathology. Thus, in recent years there has been major growth in PD biomarker research , aiming to allow the development of new diagnostic measures and new treatment strategies.

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