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.
Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the geneticdisease.
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.
The trial demonstrated a 42 percent reduction in combined cardiovascular death and recurrent hospitalizations at Month 30, with cardiovascular-related hospitalizations reduced by half. BridgeBio has been highly active in the geneticdiseases space. Participants were randomized to receive acoramidis or placebo over 30 months.
CF is a progressive geneticdisease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. In clinical trials, Alyftrek demonstrated non-inferiority to Trikafta, showing similar improvements in lung function and a reduction in sweat chloride levels.
Prime Medicine has been given the go-ahead by the FDA for the first human trial of its prime editing technology, which promises to deliver one-shot therapies for a range of severe geneticdiseases.
Pharmaceutical companies and biotechs are also adapting their approaches, launching patient finding and engagement programmes that can start years before clinical trials 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 clinical trial of Jotrol in Parkinson’s disease.
It is based on data from a cohort in a Phase III, 24-week, open-label trial assessing the safety, pharmacokinetics and pharmacodynamics of ivacaftor in CF patients with an ivacaftor-responsive CFTR mutation who are under 24 months old. This cohort showed a safety profile identical to that found in older children and adults.
TSHA-102 is the first gene therapy in the clinical development stage for Rett syndrome currently while TSHA-120 is being analysed in a Phase I/II clinical trial for treating GAN. Astellas will also receive specific rights linked to any possible change of Taysha’s control.
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. Crenessity marks the third approved drug for Neurocrine.
The Japanese pharma contends that an analysis of the four deaths in its AT132 gene therapy clinical trial 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 clinical trials and real-world settings. Late in 2024, Elevidys was recognized among Times Best Innovations. million.
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 clinical trial portfolio. As such, rare disease patients and their families often face little hope for effective treatments.
CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. Ultragenyx Pharmaceuticals has an orthinine transcarbamylase (OTC) activator drug in Phase III trials.
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 clinical trials.
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.
The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare geneticdiseases. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on. .
In rare diseasetrials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Verifying the biomarker’s clinical validity for use as a surrogate endpoint in rare disease research is another hurdle which is generally a longer-term goal.
Axovant has said it plans to continue developing its Parkinson’s Disease gene therapy after reporting supportive data from a small cohort of patients from a phase 2 trial. The data came from a second cohort of just four patients in the phase 2 clinical trial SUNRISE-PD, although the improvement in symptoms was dramatic.
A gene therapy being developed by Lexeo Therapeutics to treat cardiomyopathy caused by Friedreich ataxia, a rare neurodegenerative geneticdisease, has achieved good interim results in a phase 1/2 trial.
The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8
A tiny child with a devastating geneticdisease who wasn’t supposed to blow out the candles on his first birthday cake. All clinical trials 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. Collaboration.
Research and development in the area is currently growing at a fast rate, and the National Institute of Health reports hundreds of clinical trials to test gene therapies for different geneticdiseases, immune system disorders, oncology treatments, neurogenerative diseases, infectious diseases, and more.
In the age of artificial intelligence, no trial data should be going to waste. Findacure’s Rick Thompson looks at how these technologies could bring us closer to treatments for underserved rare diseases. The repurposing of drugs is becoming more common, especially in the field of rare diseases.
Clinical trials 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 clinical trials and drug development about benefit,” Dr Mullen said. But who is benefitting?
With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare geneticdiseases with a focus on blinding disorders of the retina.
This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year. Related: World Thrombosis Day 2024: Move Against Thrombosis This approval stems from the results of the Phase III BASIS trial, which evaluated the efficacy and safety of Hympavzi in patients with hemophilia A or B.
The regulatory nod “ushers in a whole new paradigm to treat geneticdiseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.
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 disease clinical trials and orphan drugs. How is a Rare Disease Defined? Are Most Rare DiseasesGenetic?
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. The majority of these are in Phase I (68%) and Phase II (24%), with just 8% in Phase III.
BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.
Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial 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.
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 clinical trials and quality of life measurements. and Leila Zegna, director of the Kabuki Syndrome Foundation.
Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare geneticdisease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial.
PKU is a rare geneticdisease that manifests at birth and is marked by an inability to break down phenylalanine (phe), an amino acid that is commonly found in many foods. Left untreated, high levels of the amino acid become toxic to the brain and may lead to serious neurological and neuropsychological issues.
Pompe disease primarily manifests as progressive muscle weakness, particularly affecting the skeletal muscles in regions such as the hips, legs, shoulders, arms and diaphragm. Sanofi also offers another ERT for Pompe disease known as Myozyme, which received FDA approval in 2006, and Lumizyme in 2010 for late-onset disease.
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.
The FDAs approval was supported by data from the Phase III RESTORE study a 24week, doubleblind, placebocontrolled, randomized crossover withdrawal trial. Administered as an oral tablet at a dosage of 250 mg three times per day, the treatment aims to restore normal bile acid levels and reestablish metabolic equilibrium.
Sarepta reported updated clinical trial results with the one-shot therapy in July which bolstered the data for SRP-9001 (delandistrogene moxeparvovec) for efficacy and durability, but also raised a concern about its safety after a serious case of myocarditis was seen in one of 38 patients enrolled in its ENDEAVOR study.
CRISPR technology has begun to enter clinical trials due to emerging therapeutic applications, but the technology still has limitations, primarily because it is difficult to safely make large quantities of precisely edited cells.
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. Advisors also had some concerns about the safety of the drug and the design of the pivotal trial used to support the marketing application.
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