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Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. The post Lilly and ProQR to expand genetic medicine development agreement appeared first on Pharmaceutical Technology.
At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.
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.
Roughly one out of three women ages 14-49 in the United States develop a vaginal bacterial imbalance known as bacterial vaginosis (BV) during their lifetime. BV is characterized by unpleasant odors, and potentially painful side effects, as well as the risk of associated health issues later in life.
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.
Fortunately, advances in clinical research are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
Fortunately, advances in clinical research are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
While several therapeutics are available for treating symptoms associated with epilepsy, researchers and patients have strongly called out the need for more holistic treatments that would address the condition as a whole. Meanwhile, others are researching the link between gene variation and different responses to treatments.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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?
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 researchersdevelop new brain cancer therapy appeared first on Pharmaceutical Technology.
While it remains unclear why some people become more gravely ill than others when infected with the SARS-CoV-2 virus that causes COVID-19, researchers have found that an immune deficiency may be part of it. Casanova’s lab is now searching for the genetic driver behind the autoantibodies. The analysis revealed that 23 (3.5
Swiss medical data specialist Sophia Genetics has launched a platform that will sift through data generated at more than 1,000 hospitals around the world to try to work out how the COVID-19 pandemic will evolve in the coming months and years. The post Sophia Genetics launches AI tool to find COVID-19 ‘unknowns’ appeared first on.
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.
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.
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?
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!
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.
Alphabet subsidiary and precision health company Verily recently announced a breakthrough in its AI drug discovery GPCR research collaboration with Sosei Heptares. Kim recognised then, he said, that better tools for medicine needed to be developed, and that they still do. Human-first precision health.
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 developinggenetic medicines for neurological disorders for specific genetic targets.
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Similarly, DNA molecules can be genetically modified and introduced into human cells, or individual patient cells can be genetically modified and reintroduced to the body. PK Planning for Gene Therapy Development Programs.
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.
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.
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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