Drug Discovery World

MARCH 18, 2024

Landmark genetic research could allow doctors to accurately predict whether a patient is at risk of developing common diseases, decades before any symptoms would become evident. The post $8 million awarded to landmark genetic research project appeared first on Drug Discovery World (DDW).

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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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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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Medical Xpress

FEBRUARY 21, 2023

Researchers from Children's Hospital of Philadelphia (CHOP) developed two new zebrafish models for studying a specific genetic form of mitochondrial disease that represents the most common cause of Leigh syndrome. The findings were recently published in the journal Human Molecular Genetics.

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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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Drug Discovery World

OCTOBER 5, 2023

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. The researchers have shown that problems with calcium underlie these progressive diseases.

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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.

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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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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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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 Innovation in Pharmaceuticals: Genetically modified animal models. Regeneron Pharmaceuticals is one of the leading patent filers in the generation of genetically modified animal models.

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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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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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Drug Discovery World

FEBRUARY 28, 2024

Lu Rahman selects some of the year’s interesting and noteworthy advances in cancer research drug discovery and development. As always, a year within cancer drug discovery and development brings with it significant breakthroughs, and 2023 has been no exception. Research into new cancer drugs remain strong.

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Pharmaceutical Technology

JUNE 4, 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 Innovation in Pharmaceuticals: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

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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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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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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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Medical Xpress

NOVEMBER 29, 2022

Genetically modified and attenuated measles viruses are considered a promising platform for research and development of vector vaccines and oncolytic measles viruses.

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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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Olympian Clinical Research

SEPTEMBER 27, 2023

While the exact cause of vitiligo remains a subject of ongoing research, genetics has emerged as a significant factor in understanding its onset and progression. In this deep dive, we’ll explore the genetic links associated with vitiligo and address the burning question: Is vitiligo hereditary?

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Drug Discovery World

JANUARY 19, 2024

UK scientists have uncovered the interplay between cancer-driving genetic mutations and inherited genetic variants in a rare type of blood cancer. In the future, this knowledge could aid drug development and interventions that reduce the risk of disease. MPNs can also progress into other forms of blood cancer, such as leukaemia.

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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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Scienmag

NOVEMBER 3, 2020

Credit: Encarnacion/UConn Photo The advent of genome science has given researchers an unprecedented ability to understand the root causes of a host of conditions.

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BioSpace

JUNE 23, 2022

The Boston University School of Medicine has unraveled details about a known genetic component for the development of Alzheimer’s disease (AD): the APOE4 gene.

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Pharmaceutical Technology

MAY 19, 2023

ImmunoForge has expanded its licence agreement with Duke University in North Carolina, US, to jointly improve research and drug development opportunities. ImmunoForge chief technology officer Dr Jim Balance pioneered the clinical development of peptide drugs genetically fused to ELPs.

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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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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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Drug Discovery World

DECEMBER 13, 2022

PacBio, developer of sequencing solutions, has announced its HiFi sequencing technology will be used in a pilot project for the Children’s Rare Disease Cohorts Initiative (CRDC) at Boston Children’s Hospital. . As causative variants are identified by CRDC researchers, results are returned to participants.

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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.

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BioTech 365

AUGUST 19, 2021

Achromatopsia (Genetic Disorders) Drugs in Development, 2021 Market Research Report – ResearchAndMarkets.com Achromatopsia (Genetic Disorders) Drugs in Development, 2021 Market Research Report – ResearchAndMarkets.com DUBLIN–(BUSINESS WIRE)–The “Achromatopsia (Genetic Disorders) – Drugs in Development, 2021” (..)

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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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pharmaphorum

MARCH 21, 2022

Neuroscience is arguably the next great frontier in medicine and 2022 dawned with a wave of exhilarating progress for the neurodegenerative drug development community. A startup led by Novartis veteran raises $80 million to develop treatments for people with degenerative brain conditions.

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Medical Xpress

APRIL 6, 2023

The risk of developing cardiovascular disease is lower in people with obesity who have a genetic predisposition for high BMI than people with obesity influenced mainly by environmental factors such as lifestyle, researchers from Karolinska Institutet report in eClinicalMedicine.

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Pharmaceutical Technology

JUNE 8, 2023

Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.

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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.

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Outsourcing Pharma

FEBRUARY 24, 2022

The pharmaceutical companyâs Lilly Institute for Genetic Medicine reportedly will focus on developing RNA-based therapeutics in a âstate-of-the-artâ facility.

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Medical Xpress

NOVEMBER 22, 2022

A team of researchers affiliated with several institutions in Alabama has linked 10 genetic loci to the development of osteoarthritis. In their paper published in the journal Nature Genetics, the group describes their analysis of data from the Million Veteran Program in the U.S. and the UK Biobank in the U.K.

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