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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.
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 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.
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.
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.
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.
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.
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.
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
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.
Sarepta reported updated clinicaltrial 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.
The technology is being deployed as a therapeutic in two ways – either modifying cells outside the body (ex vivo), which are reinfused to treat a disease – or directly administered (in vivo) to target tissues in the body.
The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinicaltrial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. Sarepta Therapeutics, Inc. www.lysogene.com.
What’s been shown in mouse models of Rett syndrome which also have the geneticdisease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.
PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinicaltrial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).
Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency.
Expect to initiate INZ-701 Phase 1/2 clinicaltrials for ENPP1 and ABCC6 deficiencies in first half of 2021.
H1 2021: Initiation of Phase 1/2 clinicaltrial.
However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinicaltrials. Late in 2019, a Fabry patient in phase 2 clinicaltrial of ex vivo lentiviral gene therapy AVR-RD-01 became the first patient dosed using Plato.
1 These results confirm the favourable safety profile of Hemlibra, as previously demonstrated in the phase III HAVEN clinicaltrials. Spark Therapeutics will share updated data from the ongoing phase I/II clinicaltrial of SPK-8011, an investigational AAV-based gene therapy developed for the treatment of haemophilia A.
Acromegaly Surrogate Endpoint: Serum growth hormone and serum insulin-like growth factor 1 (IGF-1) are acceptable surrogate endpoints for acromegaly clinicaltrials involving somatostatin analogs such as octreotide, lanreotide and pasireotide. The traditional approval pathway can be pursued by meeting this surrogate endpoint.
Safety is critical in all clinicaltrials, but with ultra-rare diseases, even greater care is taken. We’ve created safety databases that begin with non-human primates and extend to controlled clinicaltrials.” Geneticdiseases are vastly more common and more complex than we used to think.”.
As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these geneticdiseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.
Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for ClinicalTrial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP. Adzynma’s approval is the second for Takeda this week.
The company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare geneticdiseases.
SparingVision has raised €75 million in second-round funding that will be used to fund clinicaltrials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD).
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