Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)

This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.

Start Date01 Nov 2016

Sponsor / Collaborator

MeiraGTx UK II Ltd.

NCT02781480 / CompletedPhase 1/2

An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)

A clinical trial of AAV2/5 vector for patients with Defects in RPE65

Start Date01 Apr 2016

Sponsor / Collaborator

MeiraGTx UK II Ltd.

100 Clinical Results associated with Cevaretigene ritoparvovec (MeiraGTx)

100 Translational Medicine associated with Cevaretigene ritoparvovec (MeiraGTx)

100 Patents (Medical) associated with Cevaretigene ritoparvovec (MeiraGTx)

Literatures (Medical) associated with Cevaretigene ritoparvovec (MeiraGTx)

01 Dec 2016·Gene TherapyQ3 · MEDICINE

Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

Q3 · MEDICINE

Article

Author: Michaelides, M ; Sünkel-Laing, B ; Abelleira-Hervas, L ; Gonzalez-Cordero, A ; Georgiadis, A ; Robbie, S J ; Bainbridge, J W B ; Fourali, S ; Ribeiro, J ; Smith, A J ; Duran, Y ; Ali, R R ; Cristante, E

AbstractLeber congenital amaurosis is a group of inherited retinal dystrophies that cause severe sight impairment in childhood; RPE65-deficiency causes impaired rod photoreceptor function from birth and progressive impairment of cone photoreceptor function associated with retinal degeneration. In animal models of RPE65 deficiency, subretinal injection of recombinant adeno-associated virus (AAV) 2/2 vectors carrying RPE65 cDNA improves rod photoreceptor function, and intervention at an early stage of disease provides sustained benefit by protecting cone photoreceptors against retinal degeneration. In affected humans, administration of these vectors has resulted to date in relatively modest improvements in photoreceptor function, even when retinal degeneration is comparatively mild, and the duration of benefit is limited by progressive retinal degeneration. We conclude that the demand for RPE65 in humans is not fully met by current vectors, and predict that a more powerful vector will provide more durable benefit. With this aim we have modified the original AAV2/2 vector to generate AAV2/5-OPTIRPE65. The new configuration consists of an AAV vector serotype 5 carrying an optimized hRPE65 promoter and a codon-optimized hRPE65 gene. In mice, AAV2/5-OPTIRPE65 is at least 300-fold more potent than our original AAV2/2 vector.

News (Medical) associated with Cevaretigene ritoparvovec (MeiraGTx)

15 Oct 2024

·endpts.com

MeiraGTx's stock rises on small Parkinson's gene therapy dataset

MeiraGTx unveiled results from a small dataset for its Parkinson’s disease gene therapy on Tuesday morning, causing its shares $MGTX to jump by as much as 24%. In what the company called a “bridging study” featuring 14 patients, the high dose of MeiraGTx’s AAV-GAD showed a statistically significant improvement on a Parkinson’s disease measurement score known as MDS-UPDRS Part 3 after 26 weeks. Five patients in the high-dose group improved their “off” state scores by an average of 18 points from baseline, good for a p-value of 0.03. MeiraGTx CEO Alexandria Forbes said in a statement that the company expected a five- to 10-point improvement to represent a clinically meaningful change. The sham group (four patients) and low-dose group (five patients) saw no significant changes compared to baseline. The primary endpoint was safety and tolerability, and was met. In another exploratory endpoint, MeiraGTx said a quality-of-life measure called the PDQ-39, which uses a patient questionnaire, achieved statistical significance in both dosing groups when compared to patients’ baseline scores. There was only a “trend to significance” between the high-dose group and the sham group, MeiraGTx said. The company is still discussing its Phase 3 plans with regulators, but expects to launch a pivotal trial in the first half of 2025, according to a corporate slide deck updated Tuesday morning. Gene therapies for Parkinson’s disease have been attempted before, though none have been FDA-approved. MeiraGTx’s program is designed to reset the abnormal brain activity associated with the disease by targeting production of a brain chemical called gamma-aminobutyric acid, or GABA. AAV-GAD attempts to deliver a functional copy of a gene called GAD into the brain that would increase GABA production and alleviate motor symptoms.

Gene TherapyClinical ResultPhase 3

15 Oct 2024

·medcitynews.com

On Heels of Positive Parkinson’s Gene Therapy Data, MeiraGTx Looks Ahead to Pivotal Test - MedCity News

A MeiraGTx Holdings gene therapy for Parkinson’s disease has preliminary data from a small clinical trial showing the one-time treatment showed both safety and efficacy, results that lay the groundwork for discussions with regulators about a Phase 3 study. The MeiraGTx gene therapy, AAV-GAD, had previously posted Phase 1 and Phase 2 results showing it was well tolerated with no significant adverse effects. The results announced Tuesday are from a Phase 1 bridging study designed to evaluate doses of AAV-GAD manufactured by London-based MeiraGTx at its own facilities using its commercial platform process. Parkinson’s patients lack sufficient amounts of a neurotransmitter called dopamine. Standard treatment includes the drug levodopa, a compound that a brain enzyme converts into dopamine. But dopamine replacement therapies lose efficacy over time. The motor symptoms of Parkinson’s can also be treated with deep brain stimulation, but that’s an invasive procedure. sponsored content Upcoming Webinar: Battle of the Budget: Employers vs. Rising Healthcare Costs Gain valuable knowledge and practical insights on how employers are addressing an increasingly complex healthcare budget landscape. By Personify Health MeiraGTx’s gene therapy is designed to spark local production of a neurotransmitter called GABA, which is intended to reprogram dysfunctional brain circuits and restore more normal activity to cells, alleviating motor symptoms. The 26-week bridging study enrolled Parkinson’s patients who had a history of responsiveness to levodopa treatment for at least 12 months. A total of 14 participants were randomly assigned to receive a high dose, a low dose, or a sham treatment. According to MeiraGTx, AAV-GAD met the main goal of safety and tolerability with no serious adverse events (SAEs) reported. Efficacy, an exploratory endpoint, was evaluated by measuring motor examination scores. The company said at week 26, results show a statistically significant 18-point average improvement from baseline according to a rating scale used to measure the severity and progression of Parkinson’s. By comparison, no significant change was observed in the low-dose group or the sham cohort. “With material made using our proprietary production process at commercial scale, we have demonstrated that AAV-GAD is safe at all doses studied, including a higher dose than previously tested,” MeiraGTx President and CEO Alexandria Forbes said in a prepared statement. “We have now treated a total of 58 patients in this development program in three independent multicenter clinical studies and have seen no SAEs related to AAV-GAD treatment.” Participants who completed the bridging study have the option of enrolling in a long-term follow-up study, which will monitor them for five years. Meanwhile, MeiraGTx said it plans to discuss the results with regulators in the U.S., Europe, and Japan as it looks ahead to pivotal testing of the gene therapy. presented by Sponsored Post The Promise of Value-Based Care and MedTech Innovation Monica Vajani, Executive Director for mHUB’s MedTech Accelerator, discusses how mHUB is helping innovators transition healthcare towards value-based care. By Monica Vajani - mHUB MeiraGTx isn’t the only company working to develop a one-time treatment for Parkinson’s. Bayer’s bemdaneprocel is made by engineering donor cells, turning them into dopamine-producing neurons. These cells are surgically transplanted into a patient’s brain where they are intended to replace a patient’s lost dopamine production. Last year, Bayer reported positive safety and efficacy data from bemdaneprocel’s Phase 1 test. Last month, Bayer reported these encouraging trends continued measured at 24 months. Aspen Neuroscience is also developing a Parkinson’s cell therapy. But unlike Bayer’s approach, Aspen’s ANPD001 is made from stem cells sourced from a patient’s own skin, offering the potential to avoid immunosuppressive drugs needed to prevent the body from rejecting the transplanted cells. Last month, Aspen announced it had completed dosing of the first cohort of a Phase 1/2a test of this cell therapy. Image: Dr_Microbe, Getty Images

Clinical ResultCell TherapyPhase 1Gene TherapyPhase 3

06 Jun 2024

·www.pharmaceutical-technology.com

Moderna and Myrtelle among those picked for FDA rare disease programme

The FDA’s pilot programme is set to accelerate the development of novel drug and biological products for rare diseases. Credit: Tada Images via Shutterstock. Moderna and Myrtelle’s rare programmes will both be included in a pilot programme by the US Food and Drug Administration (FDA) that aims to accelerate the development of novel drugs and biological products for rare diseases. Recommended Buyer's Guides Buyer's Guide Top Guide for Drug Delivery Systems Buyer's Guide Leading Guide to Clinical Packaging Companies in contract Manufacturing for the Pharmaceutical Industry Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. Thank you. You will receive an email shortly. Please check your inbox to download the Report. Go deeper with GlobalData Reports LOA and PTSR Model - Gene Therapy For Melanoma And Multiple Myeloma... Reports NPVM: MeiraGTx Holdings Plc's Cevaretigene Ritoparvovec Premium Insights The gold standard of business intelligence. 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Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Announced in September 2023, the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) programme aims to expedite new drug development by providing sponsors with frequent advice and regular communication with the FDA to advance their programme as quickly as possible. Moderna’s START programme evaluates mRNA-3705, an investigational therapeutic for methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase (MUT) deficiency. The candidate is being investigated in a Phase I/II Landmark study (NCT04899310), designed to evaluate the safety and tolerability of Mrna-3705 administered via intravenous infusion (IV) in patients one year and older with isolated MMA due to methylmalonyl-CoA mutase (hMUT) deficiency. Myrtelle is developing rAAV-Olig001 for Canavan disease (CD), a fatal childhood genetic disorder characterised by the collapse of white matter in the brain due to a mutation in the aspartoacylase gene (ASPA). This affects the creation of myelin, leading to a deficiency in the aspartoacylase enzyme. The gene therapy is a new vector from a class of recombinant adeno-associated viruses (rAAVs) that selectively target oligodendrocytes, the cells in the brain that produce myelin. See Also: The tiny pills that may send shockwaves across immunology Roche’s PI3K inhibitor secures breakthrough status in breast cancer It is being evaluated in a Phase l/ll clinical trial (NCT04833907) enrolling 24 patients with CD. It involves administering the gene therapy to patients using neurosurgical procedures, which involves direct administration of gene therapy to affected regions of the brain. Outcome measures for the open-label clinical trial include longitudinal clinical assessments and brain imaging. In the announcement accompanying the acceptance, Myrtelle global regulatory affairs senior vice president Nancy Barone Kribbssaid: “Opening the lines of communications beyond traditional meeting pathways provides the opportunity to quickly address development issues that would otherwise delay progression to market application.” Applicants had to meet certain criteria from the Center for Biologics Evaluation and Research (CBER), which has selected four participants for the pilot programme;, and the Center for Drug Evaluation and Research (CDER), which has selected three. Earlier this week, Denali Therapeutics , Neurogene, Larimar Therapeutics and Grace Science all announced that programmes will be included in the pilot. Neurogene’s gene therapy NGN-401 is currently under investigation to treat Rett syndrome. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. Thank you. You will receive an email shortly. Please check your inbox to download the Report. 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Gene TherapyClinical Study

100 Deals associated with Cevaretigene ritoparvovec (MeiraGTx)

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Indication	Highest Phase	Country/Location	Organization	Date
Leber Congenital Amaurosis	Preclinical	GB	University College London	09 Mar 2016

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02781480 (OPTIRPE65, CTgov)	Phase 1/2	Retinal Dystrophies \| Amaurosis Congenita of Leber, Type 1	15	AAV RPE65 (Low Dose AAV-RPE65)	noojveyyqb(vremcukkme) = zhsfdxcyxm aleejzuvtm (eigwgaecym, zqyywvjfdj - tdojfjdyok) View more	-	08 Apr 2021
				AAV RPE65 (Intermediate Dose AAV-RPE65)	noojveyyqb(vremcukkme) = ehwfkeafdg aleejzuvtm (eigwgaecym, ubznysimdy - obnhwbknmi) View more

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