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.

Genetics 114

Genetics Research Genome Genomics 114

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!

Genetics 130

Genetics Clinical Trials Clinical Trials Genotype 130

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.

Gene Therapy 305

Gene Therapy Gene Development Genetics 305

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.

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

Pharma Mirror

APRIL 28, 2021

Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinical trials in the U.S. and Europe. Catalent will further support process optimization and look to reduce material.

Gene Therapy 130

Gene Therapy Genetic Disease Genetics Gene 130

Pharmaceutical Technology

JUNE 15, 2023

But before pharmaceutical companies can go to market with a breakthrough drug, they need to ensure safety and efficacy through clinical trials. Pharma R&D teams are solving this problem by leveraging the power of artificial intelligence (AI) in clinical trials to save time and money. Food and Drug Administration approval.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Development 130

STAT News

DECEMBER 10, 2022

NEW ORLEANS — An emerging class of genetically targeted drugs is inducing remissions in about one-third of patients with advanced leukemia, according to updates Saturday from separate clinical trials. Continue to STAT+ to read the full story…

Genetics 141

Genetics Drugs Clinical Trials Clinical Trials 141

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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Development Gene Therapy Protein Gene 246

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.

Vaccination 307

Vaccination Vaccine Development Clinical Trials 307

Pharmaceutical Technology

JUNE 10, 2023

CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

Genetic Engineering 100

Genetic Engineering Engineer Genetics Clinical Trials 100

Pharmaceutical Technology

JUNE 10, 2023

CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

Genetic Engineering 100

Genetic Engineering Engineer Genetics Clinical Trials 100

Roots Analysis

JANUARY 19, 2024

Introduction In a strategic move to accelerate the development and launch of genetic medicines, BridgeBio Pharma has successfully secured up to USD 1.25 Billion to Propel Genetic Medicine Development appeared first on Blog. billion from Blue Owl Capital and Canada Pension Plan Investment Board.

Genetics 40

Genetics Medicine Development Sales 40

FDA Law Blog

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

Genetics 59

Genetics Production Development Drugs 59

Pharmaceutical Technology

DECEMBER 12, 2022

Gilead company Kite has entered an international strategic partnership with Arcellx for the joint development and commercialisation of the latter’s T-cell therapy, CART-ddBCMA, to treat relapsed or refractory multiple myeloma patients. Additionally, Kite will make other potential payments to Arcellx.

Development 264

Development Gene Therapy Clinical Trials 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. The post Capsida Biotherapeutics and Prevail to develop CNS gene therapies appeared first on Pharmaceutical Technology.

Gene Therapy 237

Gene Therapy Gene Development Engineer 237

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Drug Discovery World

APRIL 15, 2024

Naobios has signed a partnership agreement with Sumagen, a Korean-Canadian biotechnology company developing an HIV-1 vaccine candidate, to manage the manufacturing process during its Phase II clinical trials. At the end of 2022, the World Health Organization (WHO) reported that approximately 39 million people were living with HIV.

Vaccination 52

Vaccination Vaccine Development Clinical Trials 52

BioPharma Reporter

MARCH 5, 2024

Neurogene, a clinical-stage company developing genetic medicines, is expanding its ongoing phase 1/2 clinical trial investigating NGN-401 as a treatment for female pediatric patients with Rett Syndrome.

Clinical Trials 59

Clinical Trials Clinical Trials Gene Therapy Trials 59

Drug Discovery World

OCTOBER 11, 2023

Gain Therapeutics has initiated a Phase I clinical trial of GT-02287, the company’s lead drug candidate for the treatment of GBA1 Parkinson’s disease. The post Allosteric therapy targets common genetic cause of Parkinson’s appeared first on Drug Discovery World (DDW).

Genetics 72

Genetics Trials Clinical Trials Clinical Trials 72

pharmaphorum

MARCH 10, 2022

18 months after signing its first partnering deal with Genentech, Scenic Biotech has raised $31 million in first-round financing that it will use to take its own genetic modifier-targeted medicines into clinical trials. The post Scenic Bio raises $31m for genetic modifier platform appeared first on.

Genetics 98

Genetics Gene Sequencing Gene Clinical Trials 98

Pharmaceutical Technology

AUGUST 16, 2022

Q32 Bio has signed a partnership and option agreement with Horizon Therapeutics for developing ADX-914 to treat autoimmune ailments. According to the deal, Horizon will provide the funding to develop the therapy until the conclusion of its two Phase II clinical trials.

Development 130

Development Clinical Trials Clinical Trials FDA Approval 130

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.

Life Science 162

Life Science Development Gene Therapy Genetics 162

XTalks

APRIL 7, 2022

UK-based molecular diagnostics start-up company Genedrive PLC has developed a genetic test that can prevent deafness in babies. The Genedrive System is the world’s first genetic swab test technique that replaces conventional genetic testing that can take up to a week for results to be available.

Genetics 98

Genetics Trials Medicine Development 98

Scienmag

FEBRUARY 18, 2022

Dignity Health in Arizona is launching the first research study in North America in which genetic testing is used to identify men and women at risk of developing heart disease based on the makeup of their DNA. If proven effective in clinical trials, this form of genetic testing may be adopted globally to prevent heart disease.

Genetics 75

Genetics DNA Clinical Trials Clinical Trials 75

Pharmaceutical Technology

OCTOBER 26, 2022

SK bioscience has signed a new collaboration agreement with the Coalition for Epidemic Preparedness Innovations (CEPI) to develop messenger ribonucleic acid (mRNA) vaccines for infectious diseases. The company has obtained the newest vaccine platform technologies such as bacterial culture, cell culture and genetic recombination.

Vaccination 130

Vaccination Vaccine Development Gene Therapy 130

Pharmaceutical Technology

JUNE 6, 2023

Alkeus Pharmaceuticals has raised $150m in Series B financing to support the registration and launch of gildeuretinol (ALK-001) to treat Stargardt disease, a genetic cause of blindness in children and young adults. The genetic cause is well characterised, but to date there is no effective treatment.

Genetics 279

Genetics Life Science Trials Clinical Development 279

Drug Discovery World

AUGUST 7, 2023

DDW’s Reece Armstrong speaks to Shantanu Dhamija , VP of Strategy and Innovation at Molecular Devices about challenges of diversity in clinical trials and why induced pluripotent stem cells might be key to representing wider patient groups. RA: Why is US clinical trial diversity at its lowest point in the last 10 years?

Development 98

Development Clinical Trials Clinical Trials Drugs 98

Drug Discovery World

DECEMBER 19, 2023

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.

Engineer 113

Engineer Trials Genetic Disease Gene Therapy 113

pharmaphorum

JANUARY 18, 2023

In 2016, scientists behind a study called the Resilience Project analysed genetic data from 589,000+ people and found 13 adults who carried genetic variants that should have resulted in serious – even deadly – childhood disease, but who were apparently healthy. First, start co-development with participants early on in the process.

Genetic Disease 76

Genetic Disease Genetics Research Genome 76

XTalks

DECEMBER 28, 2023

BillionToOne, a company specializing in molecular diagnostics, has partnered with Janssen Research & Development, LLC, a Johnson & Johnson division, to conduct the AZALEA Phase III clinical trial globally. The trial is set to occur across the US, the European Union (EU) and selected global locations.

Trials 97

Trials Clinical Trials Clinical Trials Antibody 97

Worldwide Clinical Trials

MAY 14, 2024

As the strategists for Worldwide Clinical Trials’ rare disease sponsors, we design customized solutions that make a difference in achieving successful outcomes. Our approach is informed by our combined 30+ years in rare disease clinical development and our time in various stakeholder roles, such as scientists, CRAs, and project managers.

Trials 204

Trials Clinical Trials Clinical Trials Research 204

XTalks

JANUARY 15, 2024

From the continuing trend of the rapid adoption of AI tools to the growing use of real-world evidence (RWE) in regulatory decision-making and a focus on enhancing data integrity, find out how clinical trials will evolve in 2024 to shape the future of medical research. percent from 2023 to 2033.

Clinical Trials 105

Clinical Trials Clinical Trials Trials Clinical Research 105

pharmaphorum

JUNE 14, 2022

Despite more treatment options being a positive, Ben Hargreaves finds that this has raised issues over single-region clinical trials, leading to the FDA rejecting certain treatments and clarifying what is required for approval. China’s ambition to become a leader in the biopharma industry has been clear in the last few years.

Clinical Trials 89

Clinical Trials Clinical Trials Trials Development 89

Drug Discovery World

MAY 17, 2024

Deepika Khedekar , Associate Centralized Clinical Trial Lead at IQVIA Inc, looks at the challenges of utilising artificial intelligence (AI) in oncology clinical research. MIT research 4 tells us a hard truth: about 95% of these trials fall short, leaving behind a trail of broken hopes for cancer patients each year.

Trials 59

Trials Clinical Trials Clinical Trials Protein 59

Camargo

DECEMBER 10, 2021

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. This means that full approval can only come from a clinical trial that assesses adult height. Voxzogo also received a priority review voucher.

Development 176

Development Genetics Production Clinical Trials 176

pharmaphorum

NOVEMBER 29, 2021

A web-based digital tool called Helix could help clinicians identify patients with prostate cancer who would benefit from genetic testing and counselling, according to researchers in the US. One asked if they could keep using it after the two-week trial because they found it helpful for their ‘grey area’ patients.

Genetics 91

Genetics Nurses Scientist Research 91