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Switching Gene Therapy On and Off with a Pill

Posted on: 2 July 2024, source: GEN
Gene therapy has a reputation as a one-time intervention that has a lifelong effect. It’s an all-or-nothing proposition. But what if it wasn’t? What if gene therapy could be dialed up or down, on a daily basis, with a simple pill? The possibility has inspired MeiraGTx to develop a riboswitch technology that is designed to allow for the precise, dose-responsive control of gene expression by oral small molecules. The riboswitch technology is just part of MeiraGTx’s work in gene therapy. The company has technologies for the optimization of adeno-associated virus (AAV) vectors and for the design of promoter sequences. Also, the company has an internally developed manufacturing platform process and several production facilities. Finally, the company has several gene therapy programs in late-stage clinical trials.
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Toddler Born Deaf Can Hear After Gene Therapy Trial Breakthrough Her Parents Call "Mind-Blowing"

Posted on: 20 May 2024, source: CBS News
One of the youngest children in the world to receive a new type of gene therapy to treat genetic deafness can now hear for the first time in her life. The family of the toddler taking part in a medical trial has called the change in their daughter "mind-blowing." Opal Sandy, now 18 months old, was born with total deafness due to a fault in the OTOF gene, which makes a protein called Otoferlin. Otoferlin enables communication between cells of the inner ear, or cochlea, and the brain. As part of a trial run by Cambridge University, Opal received an infusion of a working copy of the OTOF gene in her right ear. The surgical procedure took only 16 minutes and was carried out just before she reached her first birthday.
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First Patient Begins Newly Approved Sickle Cell Gene Therapy

Posted on: 6 May 2024, source: The New York Times
A 12-year-old boy in the Washington, D.C., area faces months of procedures to remedy his disease. “I want to be cured,” he said. On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments.
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FDA approves Orchard’s Lenmeldy gene therapy for MLD

Posted on: 19 March 2024, source:
The US Food and Drug Administration (FDA) has granted approval to Orchard Therapeutics‘ Lenmeldy (atidarsagene autotemcel) gene therapy for use in children with specific forms of metachromatic leukodystrophy (MLD). This is the first FDA approval for a gene therapy addressing pre-symptomatic late infantile, early juvenile or early symptomatic early juvenile MLD. Lenmeldy is a single-use, personalised infusion, utilising the patient’s genetically modified hematopoietic stem cells (HSCs) to halt disease progression. The gene therapy involves collecting HSCs from the patient and modifying them to include functional copies of the ARSA gene. The modified cells are re-introduced into the patient’s bone marrow where they engraft and produce immune cells that can break down sulfatides, the substances that accumulate harmfully in MLD.
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Team performs the first gene therapy tests in a whole human liver

Posted on: 14 March 2024, source:
In a worldwide first-of-its-kind study published in the journal Nature Communications this week, a team of scientists from Children's Medical Research Institute (CMRI) have tested novel gene therapies in a whole human liver, with the goal of developing more effective treatments for life-threatening inherited diseases.
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Gene Therapy Success in Hereditary Angioedema Patients

Posted on: 2 February 2024, source: Technologynetworks
A single dose of a CRISPR/Cas9 therapy has been successful in treating hereditary angioedema. A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher. The patients from New Zealand, the Netherlands and the UK have hereditary angioedema, a genetic disorder characterised by severe, painful and unpredictable swelling attacks. These interfere with daily life and can affect airways and prove fatal.
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FDA Approves CRISPR Gene Therapy to Treat Blood Disorder

Posted on: 24 January 2024, source:
The U.S. Food and Drug Administration (FDA) has approved CRISPR gene editing cell therapy to treat a rare inherited blood disorder called transfusion-dependent beta-thalassemia (TDT). CRISPR Therapeutics and Vertex Pharmaceuticals’ gene editing cell therapy, branded as Casgevy, has officially been approved to treat TDT — a rare inherited blood disorder that requires regular blood transfusions — in patients 12 and over. Vertex announced Tuesday that it had been given FDA approval more than two months ahead of its original expected action date, and just over a month after the same treatment to treat sickle cell disease was approved.
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FDA Signals Imminent Approval for Sarepta’s Duchenne Gene Therapy: A Major Breakthrough for Patients

Posted on: 12 January 2024, source:
The world of gene therapy is on the cusp of a significant milestone. Peter Marks, the Food and Drug Administration’s (FDA) top regulator of gene therapies, has indicated the full approval of Sarepta’s Duchenne gene therapy, Elevidys, is imminent. This development has the potential to positively impact patients, caregivers, and the healthcare industry as a whole.
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Revolutionary gene therapy shows promise in treating autism

Posted on: 6 December 2023, source:
Chinese scientists have achieved a major advancement in gene therapy for autism, as reported in the journal Nature Neuroscience. This innovative therapy, developed by a research team in Shanghai, has been successfully tested on mice. The research marks a significant step in the treatment of autism spectrum disorder (ASD) through genetic base editing within the brain.
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Cell & Gene Therapy Space Gears Up for First CRISPR Approval

Posted on: 13 November 2023, source: Biospace
Halloween brings to mind pumpkins and trick or treat, but for the 100,000 Americans suffering from sickle cell disease, this past October 31 had a different meaning when an FDA advisory panel deemed the first potentially curative SCD therapy safe enough for clinical use, bolstering hopes of an FDA approval in December. It would also be the first-ever approval for CRISPR, the genetic modification technique that won its discoverers the Nobel Prize in 2020 for their 2012 breakthrough.
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Gene therapy restores hearing in children with rare form of deafness

Posted on: 28 October 2023, source: Science
Several deaf children can hear after receiving gene therapy—a first for the approach—a team at Fudan University reported today at a meeting in Belgium. The children were born deaf because they inherited two defective copies of the gene for a protein called otoferlin that helps the inner ear’s hair cells transmit sound to the brain. In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears. Four of five patients treated now have some hearing, the MIT Technology Review reports.
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UK biotech AlveoGene launches with plans for inhaled gene therapy

Posted on: 16 September 2023, source: BiopharmaDive
The company aims to capitalize on research by the Respiratory Gene Therapy Consortium, and is working first on a treatment of alpha-1 antitrypsin deficiency. The unusual approach could be a way to more reliably deliver gene therapies directly to the epithelial cells of the lungs. The company said preclinical research suggests its technology — dubbed InGenuiTy — could work with high efficiency and long-lasting effects. AlveoGene aims to bring its first candidate, a treatment for patients with alpha-1 antitrypsin deficiency or AATD, into clinical testing over the next two to three years. The company said it will also look to combine its platform with other technologies to target additional diseases such as lung surfactant deficiencies and idiopathic pulmonary fibrosis.
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Gene Therapy Targets Chronic Pain

Posted on: 28 August 2023, source: Medgadget
Scientists at New York University have developed a gene therapy for chronic pain. The technology works by targeting the NaV1.7 sodium ion channel present on neurons, which is an important component of the pain response. The researchers encoded a version of a peptide that allows a modulatory protein, called CRMP2, to bind to NaV1.7 sodium ion channels and modulate their activity. Treating neurons so that they now express this peptide interfered with the ability of CRMP2 to affect the sodium channel, reducing the transmission of pain. As chronic pain affects a large number of patients, new treatments such as this could be set to make a real difference in many lives.
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Gene therapy eyedrops restored a boy's sight. Similar treatments could help millions

Posted on: 26 July 2023, source: Albuquerque Journal
Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye. Antonio, who's been legally blind for much of his 14 years, can see again. The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio's eyes?
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Gene therapy approved by FDA for certain boys with rare genetic condition

Posted on: 10 July 2023, source: NBC Chicago
A new gene therapy treatment gained FDA approval last month to treat boys ages four and five with Duchenne muscular dystrophy, a rare and deadly genetic disorder that leads to muscle degeneration. Connor Stoll from Chicago was the first patient in the nation to receive the infusion as part of a clinical trial at Nationwide Children’s Hospital in January 2018.
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FDA Needs More Cell, Gene Therapy Mid Managers to Speed Reviews

Posted on: 8 June 2023, source: BloombergLaw
The FDA is struggling to hire mid-level managers in cell and gene therapies as the agency looks to fill more than 100 slots in this burgeoning field and remedy slow responses to drugmakers.
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Biology Professor Venigalla Rao Makes Breakthrough on Gene Therapy Research

Posted on: 31 May 2023, source: Catholic University
A new gene therapy technology being developed on campus could open the doors to healing and curing a variety of ailments, according to new research by Biology Professor Venigalla Rao published May 30 in the international scientific journal Nature Communications. Rao is the founding director of The Catholic University of America’s Bacteriophage Medical Research Center devoted to researching the therapeutic potential of a type of virus called bacteriophage T4 that grows on E.coli bacteria that cannot infect humans and many of which are part of a healthy body’s microbiome.
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Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS)

Posted on: 2 May 2023, source: MSMDS community
Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS) is an ultra rare disease caused by a mutation in the gene ACTA2 that results in a malfunction of the smooth muscle cells in the whole body. Fixed and congenital dilated pupils, patent ductus arteriosus/aorto pulmonary window and other complications related to the bladder, lungs and guts, are the common manifestations of this mutation with only 60 diagnosed people in the world, most of them children. Join the 2023 MSMDS Conference taking place May 5-7 in Boston and online.
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Bacterial ‘Nanosyringe’ Could Deliver Gene Therapy to Human Cells

Posted on: 1 April 2023, source: Scientific American
This novel injection system could help advance gene therapy by nimbly inserting gene-editing enzymes into a variety of cell types. Inside the gut of a caterpillar lives a worm, and inside the worm lurks a bioluminescent bacterium named Photorhabdus asymbiotica, which makes the caterpillar glow in the dark. But this nesting-doll-like setup has another, more harmful effect: the bacteria secrete a deadly molecular syringe, 100 nanometers long, that latches onto the insect’s cells. Once attached to a cell, the syringe pushes a molecular spear through the cell’s membrane that releases a toxic payload. As its insect host dies and decomposes, the bacteria escape to colonize their next victim.
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EU approves Hemgenix, one-time gene therapy for hemophilia B

Posted on: 7 March 2023, source: Hemophilia News Today
The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia B. Hemgenix, originally developed by uniQure, is approved for adults with severe and moderately severe hemophilia B without a history of inhibitors. It’s the first gene therapy for hemophilia B available to patients across all European Union member states, plus Iceland, Liechtenstein, and Norway.
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FDA officials offer advice on gene therapy trials

Posted on: 9 February 2023, source: Regulatory News
Officials from the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) asserted that the duration of clinical trials for gene therapies depends on the nature of the disease being treated, and that diseases that are more progressive and have a rapid onset may involve shorter trials. This was one of the learnings imparted by officials during a 7 February virtual town hall meeting to answer stakeholder questions on the clinical development of gene therapies for rare diseases outside the hematology and oncology space. The town hall is part of a series to address questions from stakeholders on topics under OTAT’s remit.
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New gene therapy delivers treatment directly to brain

Posted on: 26 January 2023, source:
When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep. Then, months later. doctors delivered gene therapy directly to her brain. Now the 4-year-old is walking, running, swimming, reading and riding horses — “just doing so many amazing things that doctors once said were impossible,” said her mother, Judy Wei.
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FDA approves Ferring’s Adstiladrin as first gene therapy for bladder cancer

Posted on: 23 December 2022, source: FDA
The US Food and Drug Administration (FDA) has approved Ferring Pharma’s Adstiladrin (nadofaragene firadenovec-vncg) as the first gene therapy for the treatment of adult patients with non-muscle-invasive bladder cancer (NMIBC). The novel adenovirus vector-based gene therapy is specifically indicated for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours and is to be administered into the patient's bladder once every three months. The FDA based its decision on a phase 3 multicentre clinical study in 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumours and could be evaluated for response.
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FDA Approves First Gene Therapy for Hemophilia B

Posted on: 29 November 2022, source: FDA
The FDA has approved etranacogene dezaparvovec for the treatment of adults with Hemophilia B who are currently using Factor IX prophylaxis therapy, have a history of life-threatening hemorrhaging, or have serious spontaneous bleeding episodes. The adeno-associated virus vector-based therapy is the first-of-its-kind for the treatment of Hemophilia B. “Gene therapy for hemophilia has been on the horizon for more than two decades,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release. “Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life.”
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FDA finalizes neurodegenerative disease gene therapy guidance

Posted on: 2 November 2022, source: Regulatory Affairs Professionals Society
The US Food and Drug Administration (FDA) last week finalized guidance to assist sponsors developing human gene therapy (GT) products for neurodegenerative diseases. In the final version, FDA acceded to industry’s requests to modify its language on the use of crossover trials, provided more detail on the use of comparability studies in assessing the effect of post-approval manufacturing changes on products and eliminated limits for residual product-related impurities.
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Gene Therapy Rapidly Improves Night Vision in Adults with Congenital Blindness

Posted on: 11 October 2022, source: Penn MedicineDNA
Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. The patients had Leber Congenital Amaurosis (LCA), a congenital blindness caused by mutations in the gene GUCY2D
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Codex DNA's BioXp 9600 System debuting at CAR-TCR Summit

Posted on: 20 September 2022, source: Codex DNA
Increasing the power of optimized synthetic biology workflows with expanded throughput and scale: Introducing the BioXpTM 9600 System. Codex DNA – a San Diego-based biotechnology company, and pioneer in automated benchtop synthetic biology systems – will be debuting their next-generation fully-automated synthetic biology benchtop workstation, the BioXp 9600 System, at this year’s CAR-TCR summit.
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BioMarin reports cancer case in hemophilia gene therapy trial

Posted on: 14 September 2022, source: Biopharmadive
Drug regulators have not ordered a trial hold after an analysis suggested leukemia was naturally occurring, company says. BioMarin Pharmaceutical, a California-based biotechnology company, said a patient in a clinical trial of its hemophilia gene therapy Roctavian has developed leukemia, although testing suggests that the case may be naturally occurring..
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Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma

Posted on: 15 August 2022, source:
Novartis has acknowledged that two patients have died of acute liver failure following treatment with its Zolgensma® (onasemnogene abeparvovec-xioi), a one-time gene therapy indicated for some forms of spinal muscular atrophy (SMA). As a result, the company said, it will revise Zolgensma’s label to specify that fatal acute liver failure has been reported.
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First patient in the Netherlands successfully treated with stem cell gene therapy

Posted on: 21 July 2022, source: LUMC
Researchers from the Leiden University Medical Center (LUMC) have successfully used stem cell gene therapy to treat a baby with the severe congenital immune disorder SCID. An important milestone: it is the first time stem cell gene therapy of Dutch origin has been administered to a patient, and also the first time it has been used to treat this particular form of SCID worldwide. The treatment was effective and the patient is doing well.
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