This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. This holds the potential to cure geneticdiseases that have plagued families for generations.
Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinicaltrials in the U.S. and Europe. Catalent will further support process optimization and look to reduce material.
Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare DiseaseClinicalTrials,” at last year’s World Orphan Drug Congress (WODC) EU. In my capstone, I focused on how, if, and when genetic counselors discuss clinicaltrials with their patients in the context of a counseling session.
CF is a progressive geneticdisease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. In clinicaltrials, Alyftrek demonstrated non-inferiority to Trikafta, showing similar improvements in lung function and a reduction in sweat chloride levels.
Pharmaceutical companies and biotechs are also adapting their approaches, launching patient finding and engagement programmes that can start years before clinicaltrials begin and allow them to run ‘recontact by genotype’ studies that the Resilience Project would have liked to do. Giving participants something in return.
In addition, preclinical studies of Jotrol in a Parkinson’s disease mouse model at the University of Miami have shown promise, addressing hallmark symptoms like nigral cell loss and dopamine deficits. Proceeds from the IPO will propel key initiatives, including the Phase II clinicaltrial of Jotrol in Parkinson’s disease.
The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinicaltrial portfolio.
Additionally, Astellas will obtain an exclusive option for licencing two clinical-stage programmes of Taysha, namely TSHA-102 and TSHA-120, for Rett syndrome and GAN, respectively. Astellas will also receive specific rights linked to any possible change of Taysha’s control.
The Japanese pharma contends that an analysis of the four deaths in its AT132 gene therapy clinicaltrial shows it is still viable as a potential treatment for a fatal, rare geneticdisease.
Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. To date, over 800 patients have received the therapy in clinicaltrials and real-world settings. Late in 2024, Elevidys was recognized among Times Best Innovations.
The Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a geneticdisease mostly affecting girls that causes severe neurologic impairments, robbing them of the ability to communicate or control muscle movement. The new drug, called Daybue, is made by Acadia Pharmaceuticals.
CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinicaltrials go forward for their urea cycle disorder programs late next year. In order to find the specific RNA sequences for a geneticdisease, the biotech is using an AI- and machine learning-based approach.
It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious geneticdisease affecting the skin and mucosal tissues. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinicaltrials.
Clinicaltrials design and patient input The definition of patient centricity, in fact, and its benefits are now – finally – being defined by patients themselves. “We We talk a lot in clinicaltrials and drug development about benefit,” Dr Mullen said. But who is benefitting?
Related: Arrowhead Pharmas New Rare Disease Campaign for FCS Highlights Importance of Lowering Triglycerides The FDAs approval of Crenessity was based on two randomized, double-blind, placebo-controlled trials in 182 adults and 103 children with classic CAH.
A tiny child with a devastating geneticdisease who wasn’t supposed to blow out the candles on his first birthday cake. All clinicaltrials rely on a well-oiled machine of motivated professionals, but nowhere is this more true than in advanced-therapy studies in rare diseases where knowledge is sparse.
Research and development in the area is currently growing at a fast rate, and the National Institute of Health reports hundreds of clinicaltrials to test gene therapies for different geneticdiseases, immune system disorders, oncology treatments, neurogenerative diseases, infectious diseases, and more.
The data came from a second cohort of just four patients in the phase 2 clinicaltrial SUNRISE-PD, although the improvement in symptoms was dramatic. Parkinson’s is not a true geneticdisease, although certain genes have been linked to an increased likelihood of onset.
Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare geneticdiseases, which currently affect an estimated 280 million patients worldwide. Cell and gene therapies are not your typical clinical supply chain.
Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare geneticdiseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.
Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare diseaseclinicaltrials and orphan drugs. How is a Rare Disease Defined? Are Most Rare DiseasesGenetic?
Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat geneticdiseases. The company then utilizes the de-identified records to improve the design of clinicaltrials and quality of life measurements. and Leila Zegna, director of the Kabuki Syndrome Foundation.
Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinicaltrials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinicaltrial hold initiated in October 2019. Novartis today announced that the U.S. SVP, Chief Medical Officer, Novartis Gene Therapies. “We
By definition, however, trial master files represent a much richer and more detailed source of data on a drug and how it performs. For instance, provided a drug has not failed a trial on safety, the side effects it caused in one population could constitute on-target effects in another.
The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare geneticdisease metachromatic leukodystrophy (MLD). He added that, “A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe geneticdiseases including MLD.
It is difficult to control where the viral vectors insert genes in the genome, and it is difficult to manufacture large quantities of clinical-grade viral vectors. Scientists have previously shown that they could replace small sections of the IL2RA gene where mutations occur in patients.
PKU is a rare geneticdisease that manifests at birth and is marked by an inability to break down phenylalanine, an amino acid that is commonly found in many foods. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.
MLD is a rare geneticdisease affecting the brain and nervous system caused by a deficiency of the enzyme arylsulfatase A (ARSA), which results in the accumulation of sulfatides (fatty substances) in cells. The transaction has proven fruitful with the milestone US approval of the gene therapy just a few months later.
Throughout the course of extensive clinicaltrials, Pombiliti and Opfolda have consistently exhibited a favorable safety profile, and patients have generally tolerated the treatment well.
Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinicaltrial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on.
Clinicaltrial results reported at this year’s European Haematology Association (EHA) congress showed that a one-time treatment with exa-cel had a significant benefit in both SCD and thalassaemia patients.
Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare geneticdiseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare geneticdiseases of obesity. BOSTON, Nov.
In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.
Zolgensma meanwhile is now approved in 45 countries, with over 2,500 patients treated globally across clinicaltrials, managed access programs, and in the commercial setting. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1
Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.
In a news release published yesterday, Pfizer announced it will voluntarily withdraw its sickle cell disease (SCD) therapy Oxbryta (voxelotor) from all markets worldwide. The company said it is also discontinuing all active clinicaltrials for voxelotor and expanded access programs around the world.
XTALKS WEBINAR: Environmental Sustainability and the Supply of Medicines for ClinicalTrials Live and On-Demand: Thursday, January 16, 2025, at 10am EST (4pm CET/EU-Central) Register for this free webinar to learn how major pharma companies are working towards environmental sustainability and reducing their environmental impact.
An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. ” Trading in Reata’s shares was halted ahead of the meeting, but the stock was down 38% pre-market this morning.
Flatt Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinicaltrials. This feedback is crucial in shaping clinicaltrials and ensuring new therapies fit seamlessly into existing treatment regimens.
ClinicalTrial Behind Spevigo. The efficacy of Spevigo (spesolimab) was determined based on the results of the Effisayil 1 double-blind, randomized, Phase II clinicaltrial. Spevigo is the first approved treatment option for GPP in adults.
ClinicalTrials. STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinicaltrial. 4, 2020 14:57 UTC. BEDFORD, Mass.–( –( BUSINESS WIRE )– Stoke Therapeutics , Inc. December 6, 2020 12:00 PM – 1:30 PM; Track: 7. About STK-001.
Few of the common adverse reactions observed in patients who underwent Keytruda and Lenvima combination in clinicaltrials are fatigue , diarrhea , musculoskeletal pain , hypothyroidism, hypertension , stomatitis , decreased appetite, rash, nausea and decreased weight. The rarity of disease – 1.36
We organize all of the trending information in your field so you don't have to. Join 21,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
189 
Let's personalize your content