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Category Archives: Genetic Therapy
Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in … – GlobeNewswire
OPGx-LCA5 will advance to the next highest dose in mid-2024 based on positive safety and efficacy data
OPGx-LCA5 is well-tolerated and demonstrated clear signs of biological activity
RESEARCH TRIANGLE PARK, N.C., March 26, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).
Based on positive safety and efficacy data from the first cohort of three adult patients, the Company will advance OPGx-LCA5 into the next highest dose. Opus anticipates initiating the next cohort mid-2024. There are also future plans to expand the study population to include subjects 13 years or older.
In the first cohort, OPGx-LCA5 has been well-tolerated and demonstrated clear signs of biological activity through 90 days, warranting continued evaluation in the next highest dose, said principal investigator Tomas S. Aleman, M.D., from the Center for Advanced Retinal and Ocular Therapeutics (CAROT), of the Scheie Eye Institute, Department of Ophthalmology of the Perelman School of Medicine, University of Pennsylvania. Moreover, early anecdotal and VR challenge test feedback is encouraging and indicates that some of the patients, who have been nearly totally blind all their lives, are now able to see and identify objects for the first time.
LCA5 is a form of an early-onset retinal degeneration that affects approximately one in 1.7 million people in the U.S. Currently, there are no approved treatments for individuals with LCA5-related vision loss.
Based on these early clinical data, were excited for the potential of OPGx-LCA5 to transform the lives of patients affected by LCA5, said Ben Yerxa, Ph.D., chief executive officer of Opus. Wed like to thank the study participants and their families and the incredible efforts of the team at the University of Pennsylvania for reaching this clinical milestone, and we look forward to progressing the trial as we continue to dose escalate.
For more information on the trial, visit clinicaltrials.gov (NCT05616793).
About OPGx-LCA5 OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5 is an early-onset severe inherited retinal dystrophy. Studies in LCA5 patients have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in nine patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.
About Opus Genetics Opus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio, including a derisked LCA5 lead program currently in a Phase 1/2 clinical trial, tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit http://www.opusgenetics.com.
Media Contact: Gina Mangiaracina 6 Degrees gmangiaracina@6degreesPR.com
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FDA Approves Third Duchenne Muscular Dystrophy Treatment in Nine Months – BioSpace
Pictured: A boy sits in a wheelchair on a beach/iStock, ChiccoDodiFC
A young biotech has its first approved product after the FDA greenlit ITF Therapeutics Duvyzat (givinostat) Thursday as the third new treatment for Duchenne muscular dystrophy since last June. The approval of Duvyzat for patients six years and older was granted to ITFs parent company, Italfarmaco.
Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of Duchenne muscular dystrophy (DMD), according to the FDAs announcement.
Duvyzat adds to list of approved treatments for families facing this devastating disease and is an important step forward in accelerating transformative treatments for everyone independent of their genetic mutation, Debra Miller, founder and CEO of CureDuchenne, said in a statement emailed to BioSpace.
Leading up to the decision, Italfarmaco reported final results from the Phase III Epidys trial, which met the primary endpoint of a change in the four-stair climb assessment from baseline to 72 weeks.
Duvyzat also showed favorable outcomes in key secondary measures, according to the company, including on the North Star Ambulatory Assessment (NSAA), a 17-item scale that measures motor function skills. On the NSAA, patients treated with the new drug saw less worsening compared to placebo after 18 months. Patients received a standard-of-care steroid regimen throughout the trial. Duvyzat was generally well tolerated, with no treatment-related severe or serious adverse events.
Craig McDonald, chair of the Department of Physical Medicine & Rehabilitation at UC Davis Health and the studys lead U.S. investigator, told BioSpace he has been quite impressed with the data on Duvyzat. McDonald, who has treated nine patients with the drug, said he has a number of patients who are still ambulatory at 16 or 17 years old. DMD patients on steroid treatment typically lose the ability to walk at around 10 to 13 years of age, McDonald noted.
Duvyzat is a small molecule that acts by inhibiting histone deacetylases (HDACs), a group of enzymes that modulate gene and protein expression in the muscle. Deregulation of HDACs is a major consequence of the lack of dystrophin associated with DMD, Matt Trudeau, head of ITF, told BioSpace in an interview ahead of the approval.
While ITF only incorporated in January 2024, Duvyzat has been in development at Italfarmaco for the past six to eight years, Trudeau said. He added that ITF has the right team in place to get the drug to patients quickly. Were bringing in a team that knows how to turn systems on quickly and move with certain purpose and urgency and passion for the patients that we hope to serve.
The new company has leveraged the rare disease talent epicenter of Boston and Cambridge, Mass., to build its team, which comprises former employees of Biogen, bluebird bio and Genzyme, companies that have proven success in the rare disease space, Trudeau noted.
While Trudeau said ITFs express purpose is to create the systems, processes and communication tools to bring Duvyzat to DMD patients, the opportunity exists to establish a rare disease business in the U.S. For the moment, though, he said ITF is laser-focused on DMD.
Theyre not the only ones paying attention to the degenerative disease. In June 2023, the FDA greenlit Sareptas Elevidys as the first gene therapy for DMD. Then, in October, Santhera Pharmaceuticals got the nod for Agamree, a novel corticosteroid that will be marketed in the U.S. by Catalyst Pharmaceuticals.
The DMD pipeline is also flush with new candidates, including Pfizers mini-dystrophin gene therapy fordadistrogene movaparvovec, for which Phase III data are anticipated in the second half of 2024, and REGENXBIOs one-time gene therapy. REGENXBIO reported positive efficacy data earlier this month from the first patient to receive a higher dose level of the treatment in a Phase I/II trial.
I think weve seen some exciting therapeutics come along under investigation in recent years, McDonald said.
Heather McKenzie is a senior editor atBioSpace. You can reach her atheather.mckenzie@biospace.com. Also follow her onLinkedIn.
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Regenxbios investigational gene therapy shows promise in Hunter syndrome – PMLiVE
Regenxbio has shared positive results from a pivotal study of its investigational gene therapy in boys aged up to five years with mucopolysaccharidosis type 2 (MPS 2), a rare genetic disease estimated to affect one in every 100,000 to 170,000 births.
The company said the results will support a regulatory submission to the US Food and Drug Administration (FDA) this year under the accelerated approval pathway.
Also known as Hunter syndrome, MPS 2 occurs when a childs body does not properly digest certain sugar molecules. When these molecules accumulate over time, they can cause cell, tissue and organ dysfunction, including in the central nervous system (CNS).
There is currently no treatment to address fatal neuronopathic CNS disease in MPS 2.
Results from the pivotal section of the phase 1/2/3 CAMPSIITE trial, presented at this years WORLDSymposium, showed that MPS 2 patients treated with Regenxbios one-time gene therapy, RGX-121, achieved decreased levels of D2S6, a key biomarker of brain disease activity, below maximum attenuated disease levels 16 weeks after administration.
Patients receiving RGX-121 demonstrated an 86% median reduction in D2S6, which Regenxbio said is approaching normal levels.
The results were consistent with data from the dose-finding phase of the study, the company said, in which the majority of patients are "exceeding expectations" in neurodevelopmental function compared to natural history data up to four years.
The new long-term follow-up of those treated with RGX-121 in the dose-finding phase also showed there was a high rate of patients who were allowed to discontinue or remain nave to standard-of-care intravenous enzyme replacement therapy.
Kenneth Mills, president and chief executive officer of Regenxbio, said: "The data from this pivotal trial supports that RGX-121 changes the course of disease by restoring the gene missing in boys with Hunter syndrome and has the potential to significantly improve vital brain function for patients living with this debilitating disease.
"We are excited about these results and working quickly to complete activities to file the biologics license application this year.
Mills added that the company has already shared results from CAMPSIITE with FDA leadership, who have confirmed that, based on the totality of the evidence, they are open to accelerated approval if supported by the review of full data.
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CRISPR gene therapy Casgevy secures approval in Europe – Pharmaceutical Technology
Vertex Pharmaceuticals and CRISPR Therapeutics have scored another regulatory approval for Casgevy (exagamglogene autotemcel) after the European Commission granted conditional marketing authorisation to the gene therapy.
The CRISPR treatment is the first gene therapy approved in Europe for sickle cell disease and transfusion-dependent beta-thalassemia (TDT).
The European Commissions decision follows the positive opinion adopted by the European Medicine Agency (EMA) in December 2023. A condition marketing authorisation is valid for one year and can get renewed annually as more clinical data gets reported.
The UK Medicines and Healthcare products Regulatory Agency (MHRA) was the first authority to approve Casgevy in November 2023. The US Food and Drug Administration (FDA) followed suit with an initial approval for sickle cell disease in December 2023, and a TDT approval in January 2024.
In all three regions, the therapy is approved for the treatment of patients aged 12 and older with recurrent vaso-occlusive crises. TDT patients who can undergo haemopoietic stem cell transplantation but do not have a human leukocyte antigen-matched related donor are also eligible for the therapy. Vertex states that the latest approval makes available the treatment option to more than 8,000 patients.
Casgevys price has been under the spotlight since gaining regulatory approvals. Vertex and CRISPR have set a price of $2.2m for the one-time treatment. The companies have not made public the price in Europe, where access will be determined based on negotiations with national authorities.
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However, Vertex stated early access for TDT patients in France has been organised ahead of national reimbursement. A total of 25 authorised treatment centres are slated to open in Europe, with three already operational.
While Vertex and CRISPR have the gene therapy market in sickle cell disease and TDT to themselves in Europe, the biotechs are competing with bluebird bio in the US. On the same day the FDA signed off on Casgevy in sickle cell disease, the agency also approved bluebird bios cell-based gene therapy Lyfgenia (lovotibeglogene autotemcel). The company already had its gene therapy Zynteglo approved to treat TDT in August 2022. Lyfgenia is priced at $3.1m while Zynteglo is priced at $2.8m.
Vertexs CEO Reshma Kewalramani said: Now our goal shifts to translating these approvals into real-world patient benefit and ensuring access and reimbursement across the globe.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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A gene therapy for chronic pain – Drug Discovery News
Julia Alterman, PhD
Assistant Professor University of Massachusetts Chan Medical School
Small interfering RNAs (siRNAs) are a new class of oligonucleotide therapeutics that degrade disease-causing mRNA. However, restrictions imposed by the blood-brain barrier and short circulation times limit the therapeutic potential of siRNAs for brain disorders. In this webinar, Julia Alterman will discuss an innovative approach to achieve potent and long-term siRNA activity in the brain using a novel chemical scaffold that enables widespread siRNA brain delivery and gene silencing.
Topics to be covered
Wednesday, October 11th, 2023 | 2:00 PM - 3:00 PM Eastern Time This webinar will be available to view live and on demand.
Speaker
Julia Alterman, PhD
Assistant Professor University of Massachusetts Chan Medical School
RNA editing catalyzed by adenosine deaminases acting on RNA (ADARs) occurs naturally at thousands of sites across the human transcriptome. This process profoundly affects gene expression and influences a wide range of human diseases from cancer to neurological disorders. In this webinar, Michael Breen will discuss the role of ADAR-mediated RNA editing in neurodevelopmental disorders and how his team develops site-directed RNA editing therapeutics.
Topics to be covered
Wednesday, April 5th, 2023 | 2:30 PM - 3:30 PM Eastern Time This webinar will be available to view live and on demand.
Speaker
Michael Breen, PhD
Assistant Professor Icahn School of Medicine at Mount Sinai
RNA editing catalyzed by adenosine deaminases acting on RNA (ADARs) occurs naturally at thousands of sites across the human transcriptome. This process profoundly affects gene expression and influences a wide range of human diseases from cancer to neurological disorders. In this webinar, Michael Breen will discuss the role of ADAR-mediated RNA editing in neurodevelopmental disorders and how his team develops site-directed RNA editing therapeutics.
Topics to be covered
Wednesday, April 5th, 2023 | 2:30 PM - 3:30 PM Eastern Time This webinar will be available to view live and on demand.
Speaker
Michael Breen, PhD
Assistant Professor Icahn School of Medicine at Mount Sinai
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A gene therapy for chronic pain - Drug Discovery News
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