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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.
Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.
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.
CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. In the first trial, 122 adults received Crenessity twice daily and 60 received placebo twice daily for 24 weeks.
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.
There are options in the future to possibly apply the worldwide research and development (R&D), manufacturing and marketing expertise of Astellas in gene therapy to AAV gene therapy development programmes of Taysha for genetic ailments of the central nervous system (CNS).
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 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 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. In the US, more than 30 million people live with a rare disease.
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. However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea.
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: 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?
First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.
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.
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.
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.
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. In their experiments, half of the T-cells gained the gene that converted them to CAR-T cells due to specific targeting of the TRAC site in the cells’ genome.
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. .
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.
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.
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.
Pompe disease, also referred to as acid-maltase disease and glycogen storage disease II, is an uncommon genetic disorder characterized by the gradual weakening of both cardiac and skeletal muscles. Crowley, executive chairman of Amicus Therapeutics, in the company’s press release.
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.
Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene. Rett syndrome is a neurodevelopmental disorder — not a neurodegenerative disease.
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.
FCS is a rare genetic disorder that prevents the body from properly breaking down triglycerides (a type of fat in the blood), leading to dangerously high levels. In clinicaltrials, it showed up to an 86 percent reduction in triglyceride levels. However, Tryngolza is not the only potential solution in the pipeline.
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.
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.
CDKL5 deficiency disorder (CDD) is a rare genetic condition caused by mutations in the CDKL5 gene, which is essential for brain development and function. 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.
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.
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.
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.
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. Trials for both Juxtapid and Repatha made use of this endpoint to gain regulatory approval.
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.
n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We It’s the cause of the mutation, not the name of the disease, that matters.”. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence.
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).
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.
NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00
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.
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.
It is estimated that 900 people are living with NPC in the US, of which approximately one-third have been formally diagnosed with this ultra-rare, relentlessly progressive and fatal neurodegenerative disease. NPC is a rare genetic disorder that leads to progressive neurological symptoms and organ dysfunction.
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.
AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited geneticdisease that causes cardiac and central nervous system dysfunction.
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