Highlights & News

MeiraGTx CEO Zandy Forbes will speak at 8:55am PT / 11:55am ET today, May 14, at the Bank of America Securities 2025 Healthcare Conference in Las Vegas. Click through for the webcast (free registration required).

MeiraGTx today announced financial and operational results for the first quarter ended March 31, 2025, and provided a corporate update. In Q1 2025, the company:

*Announced strategic collaboration with Hologen AI, including a $200 million cash upfront payment to MeiraGTx and the formation of a joint venture, Hologen Neuro AI Ltd, with a further $230 million in capital committed to initially focus on expediting Phase 3 clinical development of AAV-GAD for Parkinson’s disease.

*Received U.S. Food and Drug Administration (FDA) Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for the treatment of Parkinson’s disease.

*Gained alignment with FDA on the ongoing Phase 2 AQUAx2 randomized double-blind, placebo-controlled pivotal study in Grade 2/3 radiation-induced xerostomia (RIX) to support a potential BLA filing.

*Announced the publication of efficacy data of rAAV8.hRKp.AIPL1 for the treatment of AIPL1-related retinal dystrophy, or LCA4, in The Lancet, demonstrating meaningful responses in 11 out of 11 LCA4 children treated. The company is filing for Marketing Authorization Approval (MAA) under exceptional circumstances with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and in discussions with the FDA around a potentially similar pathway to approval in the U.S.

Click through for the full press release and further details on the above highlights and more.

“A new gene therapy from publicly traded company MeiraGTx has managed to do something unprecedented: It’s given eyesight to 11 children who were born blind,” writes Alex Knapp of Forbes in a piece on MeiraGTx’s investigational gene therapy for AIPL1-associated severe retinal dystrophy. The article also notes MeiraGTx’s work on potential treatments for Parkinson’s Disease, obesity, xerostomia, and more. 

Click through for the full article (subscription / registration may be required).

“Little Harvey Haines has been given a new lease of life,” Newsweek’s Ian Randall writes, in a piece on MeiraGTx’s treatment of AIPL1-related retinal dystrophy in children, as detailed in a paper in the medical journal The Lancet earlier this week.

“Born with a rare eye condition severely affecting his sight, he struggled to communicate with children his own age, shied away from environments that he wasn’t confident in and even found it difficult to play with his siblings. But all that has changed for him and a group of other children following a revolutionary new gene therapy breakthrough.”

Click through to read the full article.

Last week, MeiraGTx announced a collaboration with Hologen AI to expedite Phase 3 development of AAV-GAD for Parkinson’s treatment — and to industrialize MeiraGTx’s proprietary manufacturing process. The deal includes $200m in upfront cash consideration at closing for MeiraGTx, plus Hologen’s $230m investment in a new joint venture, Hologen Neuro AI, to develop MeiraGTx’s AAV-GAD program, as well as other pipeline products, through to commercialization. Hologen will also fund a portion of MeiraGTx’s manufacturing operations and own a minority stake in MeiraGTx’s manufacturing subsidiary.

Read more about the collaboration, and MeiraGTx’s progress to date on its Parkinson’s program, in James Waldron’s recent piece in Fierce Biotech. 

Click through for the full article, and see the Highlights & News section of the MeiraGTx website for the full press release.

MeiraGTx CEO Zandy Forbes spoke on the panel, “Rare & Neuro Diseases” this morning, May 6, at the USA-India Chamber of Commerce’s 19th Annual BioPharma & Healthcare Summit 2025 in Boston. Click through for more information about the panel and the event, and see the “Programs & Pipeline” section of the MeiraGTx website for more detail on MeiraGTx’s work on rare and neurological disorders.

MeiraGTx CEO Zandy Forbes took part in a fireside chat today, April 29, at the Chardan event, “Data & Regulatory Catalysts — Navigating an Evolving Landscape.” Click through for the webcast (free registration).

MeiraGTx today announced a broad strategic collaboration with Hologen Limited, a world-leading developer of multi-modal generative AI foundation models of real-world clinical data for clinical medicine and pharmaceutical drug development. 

MeiraGTx will receive $200 million in upfront cash at closing, and MeiraGTx and Hologen are forming a joint venture called Hologen Neuro AI Ltd. In addition to the $200 million upfront payment to MeiraGTx, the joint venture will be funded with committed capital of up to $230 million from Hologen to fully finance the development of AAV-GAD for the treatment of Parkinson’s disease through to commercialization, as well as funding earlier stage clinical programs in the CNS, including AAV-BDNF for genetic obesity. Hologen will own a minority stake in MeiraGTx’s manufacturing subsidiary and will contribute to the annual funding of the manufacturing subsidiary.

Click through for the full press release, including more information on Hologen AI and MeiraGTx’s manufacturing capabilities, its work to date on Parkinson’s disease, and more.

MeiraGTx today announced the publication of results from the first in-human interventional study to treat children with AIPL1-associated severe retinal dystrophy. The data were published in The Lancet in a paper titled, “Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study.”

The paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally with rAAV8.hRKp.AIPL1 with similarly remarkable effect on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.

Click through to read more about MeiraGTx’s work with AAV-AIPL1 — and for links to the press release, accompanying webcast, and The Lancet publication.

The Foundation Fighting Blindness podcast, “Eye on the Cure,” recently interviewed Dr. Michel Michaelides about his work on MeiraGTx’s investigational gene therapy for AIPL1-associated severe retinal dystrophy.

Last month, The Lancet published data from four patients treated unilaterally with the investigational gene therapy, with MeiraGTx announcing follow-on positive data from a further seven patients treated bilaterally. All 11 of the 11 children treated were blind at birth and now have visual acuity in the treated eyes. The study was based on a collaboration between MeiraGTx, Moorfields Eye Hospital, London, and UCL Institute of Ophthalmology.

Dr. Michaelides is Head of Clinical Ophthalmology at MeiraGTx and a founding member of the company. He is a Consultant Ophthalmologist at Moorfields Eye Hospital in the departments of Medical Retina, Inherited Eye Disease, and Paediatric Ophthalmology, and Professor of Ophthalmology at the UCL Institute of Ophthalmology.

Click through to hear the full interview, and see our Highlights and News section for more information about the program and results.

“Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx’s investigational gene therapy, data that are prompting the biotech to seek accelerated approval in AIPL1-associated severe retinal dystrophy,” writes Darren Incorvaia of Fierce Biotech. The piece recaps MeiraGTx’s recent announcement of publication of results in The Lancet from the first in-human interventional study to treat children with AIPL1-associated severe retinal dystrophy. 

Click through for the full article, which includes links to The Lancet publication and MeiraGTx’s announcement. 

“Toddlers who were born with the most severe form of childhood blindness can now see after groundbreaking gene therapy treatment at an NHS hospital,” writes Jada Bas of The Daily Mail, in a piece on MeiraGTx’s treatment of AIPL1-related retinal dystrophy in children, as detailed in a paper in the medical journal The Lancet this February 2025. “It is the first effective treatment in the world for the most severe form of childhood blindness and only takes an hour.”

Click through for the full article, which includes before-and-after photos and videos of patients from the study, and see the Programs & Pipeline section of the MeiraGTx website for further details on our company’s work on AIPL1-related retinal dystrophy in children.

MeiraGTx was featured in The Telegraph after announcing data on the first in-human interventional study to treat children with AIPL1-associated severe retinal dystrophy.

The study was based on a collaboration between MeiraGTx, Moorfields Eye Hospital, London, and UCL Institute of Ophthalmology.

Last week, The Lancet published data from four patients treated unilaterally with the investigational gene therapy in the study, with MeiraGTx announcing follow-on positive data from a further seven patients treated bilaterally. All 11 of the 11 children treated were blind at birth and now have visual acuity in the treated eyes.

Click through for the full article, which includes video of AIPL1 patients and quotes from patients’ families (free registration may be required). 

“[A]fter doctors injected healthy copies of the gene into their eyes with keyhole surgery that took just 60 minutes, four children can now see shapes, find toys, recognise their parents’ faces, and in some cases, even read and write,” writes Andrew Gregory of The Guardian, in a piece on MeiraGTx’s first in-human interventional study to treat children with AIPL1-related retinal dystrophy.

Click through to read the full article, and for a link to the full findings from the study, published in The Lancet medical journal this February 2025.

The published paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally, demonstrating similarly remarkable effects on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.

“A Connecticut boy is among four children all born with severe childhood blindness who gained ‘life-changing improvements’ to their vision after an experimental trial of gene therapy,” writes Vanessa Etienne of People Magazine, in a piece on MeiraGTx’s first in-human interventional study to treat children with AIPL1-related retinal dystrophy.

Data from the study were published in The Lancet medical journal in February 2025. The published paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally, demonstrating similarly remarkable effects on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.

Click through to read the full People Magazine piece, which details one young patient’s and his family’s experience with the treatment, and which links to the full publication in The Lancet.

“An experimental trial of gene therapy has helped four toddlers — born with one of the most severe forms of childhood blindness — gain ‘life-changing improvements’ to their sight,” writes Smitha Mundasad of the BBC, in a piece on MeiraGTx’s first in-human interventional study to treat children with AIPL1-related retinal dystrophy.

Data from the study were published in The Lancet medical journal this week. The published paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally, demonstrating similarly remarkable effects on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.

Click through to read the full BBC piece, which details one patient’s and his family’s experience with the treatment.

“MeiraGTx on Friday said it intends to submit a marketing authorization application (MAA) to the UK Medicines and Healthcare products Regulatory Authority (MHRA) for its investigational gene therapy for Leber congenital amaurosis 4 (LCA4), known as rAAV8.hRKp.AIPL1, based on data from 11 treated children,” writes Precision Medicine Online, in a piece on MeiraGTx’s treatment of AIPL1-related retinal dystrophy in children, as detailed in a paper in the medical journal The Lancet this February 2025.

Click through for the full article, which includes a link to The Lancet publication. The published paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally, demonstrating similarly remarkable effects on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.

Did you know that there are 25-30 million Americans, and close to 300 million people worldwide, living with rare diseases?

MeiraGTx is proud to be a part of the community of people working towards finding cures to severe, rare diseases like Leber Congenital Amaurosis.

MeiraGTx’s work on AIPL1-associated retinal dystrophy was recently published in The Lancet and featured in the Newsweek piece linked from this post.

See previous posts for more detail on Meira’s work on AIPL1 and The Lancet publication.

#ShowYourStripes on Rare Disease Day 2025.

The U.S. Food and Drug Administration (FDA) has granted MeiraGTx Rare Pediatric Disease Designation for its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1).

This is the fourth Rare Pediatric Disease Designation MeiraGTx has received in the last three months — all four for inherited retinal diseases (IRDs).

Click through to the full press release for more information. 

MeiraGTx announced today that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV2-hAQP1 for the treatment of Grade 2/3 radiation-induced xerostomia.

“This RMAT designation underscores the strength of our data indicating the potential of our AAV2-hAQP1 therapy to significantly improve the lives of patients who suffer from xerostomia following radiation treatment,” said Dr. Zandy Forbes, MeiraGTx CEO.

The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising Regenerative Medicine therapeutic candidates, including human gene therapies, that treat, modify, reverse, or cure serious or life-threatening diseases.

Click through to the full press release for more information. 

Today MeiraGTx announced Q3 2024 financial and operational results, along with recent business updates.

Key highlights from the quarter include MeiraGTx’s:

*Receipt of 3 Rare Pediatric Disease Designations (RPDD) from the FDA (U.S.) for each of 3 potential therapies for 3 different rare inherited retinopathies, including AAV-AIPL1;

*Agreement on a pathway with MHRA (UK) for Marketing Authorization Application (MAA) under exceptional circumstances for AAV-AIPL1 for the treatment of Leber congenital amaurosis (LCA4) retinal dystrophy without further clinical studies; and

*Announcement of positive data from randomized, sham-controlled clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease.

Click through to the full press release for more information. 

MeiraGTx was featured today at Fierce Biotech’s virtual event, “Advanced Therapies: Cell & Gene, mRNA, and Beyond.”

Our CEO Zandy Forbes appeared on the panel, “Cell & Gene Therapy Pulse Check: Overcoming Infrastructure and Payer Concerns.”

Click through to learn more and access video from the event. (Free registration.) 

Don’t miss BioCentury’s article, “Dynamic Range: The Key to MeiraGTx’s Quest to Control Genes with Pills,” for a close look at MeiraGTx’s work on gene expression and its innovative riboswitch technology.

“As MeiraGTx assembles its suite of technologies to control gene expression with oral pills, it has homed in on dynamic range as the element that will allow it to succeed where others have failed. Having built tailored, small molecule-activated riboswitches into its gene therapies, the company says it has achieved lower baselines and higher maximum expression than past attempts at gene control, opening the door to precision dosing of any biologic in vivo.”

“This preclinical platform is the last pillar of optimization in the clinical-stage company’s toolbox, according to CEO Alexandria (Zandy) Forbes. ‘There’s one major technology that has never been achieved, and that is to control gene expression with an oral pill,’ she told BioCentury.”

Click through for the full article.

MeiraGTx CEO Zandy Forbes appeared recently on The Business of BioTech podcast and videocast with host Matt Pillar. The episode, “Risk Mitigation with MeiraGTx’s Zandy Forbes, PhD,” goes in-depth on Dr. Forbes’s career across academic research and biotech investing as well as her founding and ongoing leadership of MeiraGTx. 

Click through for the full interview. (Free subscription log-in required.)

Fierce Biotech published a piece detailing MeiraGTx’s recent data from our clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease: “MeiraGTx’s Gene Therapy Improves Motor Function and Quality of Life in Phase 2 Parkinson’s Trial.”

The randomized, double-blind, sham-controlled study is one of three consecutive clinical trials in which 58 patients have been treated with no serious adverse events (SAEs) associated with AAV-GAD, an investigational genetic medicine.

Parkinson’s disease is a severe neurodegenerative disorder that affects more than 10 million people worldwide.

Click for the full article.

MeiraGTx today announced that the company will exhibit five posters at the European Society of Gene and Cell Therapy (ESGCT) 2024 Annual Congress in Rome, Italy, October 22 through October 25.

The posters — which range in subject matter from riboswitch-regulated gene and cell therapy to the evolution of MeiraGTx’s upstream manufacturing platform — highlight the breadth of MeiraGTx’s novel genetic medicine and cell therapy platforms.

Click for the full press release and links to the poster presentations.

MeiraGTx today announced positive data from its clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease.

The randomized, double-blind, sham-controlled study is one of three consecutive clinical trials in which 58 patients have been treated with no serious adverse events (SAEs) associated with AAV-GAD, an investigational genetic medicine. Data show significant and clinically meaningful improvements from baseline for key efficacy endpoints.

Parkinson’s disease is a severe neurodegenerative disorder that affects more than 10 million people worldwide.

Click through for the full press release and data as well as further context from MeiraGTx CEO Zandy Forbes. 

Be sure to catch this highlight from MeiraGTx CEO Zandy Forbes’s appearance at the recent World Medical Innovation Forum in Boston: Dr. Forbes’s explanation of the connection between manufacturing and our company’s vision for the future.

Click for the video segment, and see our other posts for more information on the conference and panel.

Today at the 2024 Society for Neuroscience Conference in Chicago, MeiraGTx announced a poster presentation sharing data on a potential treatment for obesity caused by MC4R genetic deficiency. The results indicate a potent, effective, locally delivered AAV-BDNF gene therapy for the deficiency in diet-induced obesity animal models.

Click through for further context from MeiraGTx CEO Zandy Forbes — and for a link to the full poster presentation.

MeiraGTx CEO and Founder Zandy Forbes joined host Rahul Chaturvedi for the Biotech 2050 podcast. In this episode, Dr. Forbes “shares her inspiring journey from academia, where she developed a deep interest in molecular biology and genetics, to her significant role in biotech investing, and eventually leading a groundbreaking gene therapy company.” The podcast also details MeiraGTx’s cutting-edge approach to genetic medicines, focusing on the development of innovative therapies for non-inherited diseases.

Click through to hear the full podcast.

MeiraGTx CEO and Founder Zandy Forbes was featured on the panel, “The Innovation Gap,” at the World Medical Innovation Forum, hosted by Mass General Brigham in Boston on Monday, September 23, 2024. The conversation explored “the future of viral vector manufacturing and the delivery of genetic medicines.”

Click through for more information on the panel and conference.

Gail Dutton of Genetic Engineering and Biotechnology News published an in-depth profile of MeiraGTx and CEO Zandy Forbes, with a special focus MeiraGTx’s riboswitch technology and details on the company’s extensive manufacturing expertise, broad clinical pipeline, and capsid/promoter optimization.

“What if gene therapy could be dialed up or down, on a daily basis, with a simple pill?” Dutton writes. “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.”

Click through to read the full piece.

At the BioCentury Grand Rounds conference this afternoon, MeiraGTx CEO Zandy Forbes will participate in the panel, “The Heyday of AAV Engineering is Here: Will It Deliver?” The event also features a poster presentation by MeiraGTx entitled “Novel Splicing Driven Gene Regulation Platform for Precise Dose Responsive Control of Gene and Cell Therapies,” about MeiraGTx’s innovative riboswitch technology.

Click through to read the full poster.

ICYMI: MeiraGTx’s genetic medicine candidate for patients suffering from xerostomia (dry mouth due to radiation damage) was recently featured at the American Academy of Oral Medicine (AAOM), where Dr. Michael Brennan (Atrium Health, North Carolina) and colleagues presented positive data from MeiraGTx’s Phase 1 AQUAx study.

Among other key findings: Patients 12 months out from treatment saw an increase in Unstimulated Whole Saliva Flow Rate of 112.8% from baseline.

MeiraGTx’s Phase 2 AQUAx2 trial is now enrolling eligible participants.

Click through to the full presentation for more details.

“MeiraGTx’s first quarter was highlighted by significant progress in our wholly-owned late stage clinical programs, including our pivotal xerostomia program and Parkinson’s program as well as our riboswitch platform,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx.

Click through to the full release for more details.

The Times UK featured MeiraGTx CEO Zandy Forbes in an in-depth profile in its Sunday Times edition. The piece covers Dr. Forbes’s journey from academic genetic research, to finance, to the founding and ongoing innovations of MeiraGTx, including our company’s riboswitch technology.

Click through for the full profile.

MeiraGTx CEO Zandy Forbes sat down with Brad Loncar at the NYSE for a segment of BiotechTV. Watch the full episode for more on our pipeline progress as well as our differentiated genetic medicine manufacturing capabilities.

Click through for the full interview.

We’re thrilled to announce an Asset Purchase Agreement with Janssen, a Johnson & Johnson company, for our gene therapy candidate for treatment of X-linked retinitis pigmentosa (XLRP). The deal positions MeiraGTx for expedited pipeline advancements, with MeiraGTx to receive $130 million in upfront and near-term milestone payments as part of the agreement, and an additional $285 million upon first commercial sales of bota-vec in U.S. and EU and manufacturing technology transfer.

Click through to the full announcement for more details.

In a recent piece in The Evening Standard (UK), Daniel Keane goes behind the scenes at MeiraGTx and spotlights our CEO, Zandy Forbes, who shares the details on our potentially revolutionary gene therapy treatments for complex diseases.

“Deep in a Shoreditch basement, a group of scientists are conducting experiments that could one day help revolutionise the treatment of blindness, cancer, and obesity. MeiraGTx, based in a former recording studio on Britannia Walk, is developing gene therapy which they hope could offer answers to some of the most complex diseases.”

Click through for the full article.

MeiraGTx today announced strategic updates, including that Sanofi has made a $30 million strategic investment in the Company through the purchase of 4.0 million ordinary shares at a price of $7.50 per share.

Paul Hudson, chief executive officer of Sanofi, said: “Our mission at Sanofi is to deliver first or best in class medicines and vaccines for patients. So, we welcome technological innovations that help us chase the scientific miracles of the future.  We believe that MeiraGTx’s Riboswitch platform, supported by its unique manufacturing capabilities, is such a technology. We look forward to working along side MeiraGTx as this innovative science moves forward to create a new generation of breakthrough medicines for immune-mediated and neurological diseases.”

Sanofi to receive a right of first negotiation (ROFN) for the use of MeiraGTx’s Riboswitch gene regulation technology for certain Immunology and Inflammation (I&I) and Central Nervous System (CNS) targets, as well as for GLP-1 and other gut peptides for metabolic disease, and for MeiraGTx’s Phase 2 Xerostomia program.

In addition, MeiraGTx has been approached by multiple parties with strategic interest in different assets of the Company and is actively pursuing these options

Click to see full press release and further details.

Media: Fierce Biotech

A piece by Helen Floersch of Fierce BioTech highlights MeiraGTx’s presentation at the European Society of Cell and Gene Therapy (ESGCT) conference in Brussels last week.

“New data from mouse and rat studies show that MeiraGTx’s gene therapy for amyotrophic lateral sclerosis, or ALS, can repair a key cell-killing process that’s common in both genetic and sporadic forms of the disease,” the article notes.

“The type of defect the treatment corrects is present in the vast majority of ALS patients, suggesting it could have wide-ranging benefit if validated in future studies.”

Click for further detail on these new findings and more in the full story.

Media: BioSpace

BioSpace’s Nadia Bey published a recent profile of MeiraGTx CEO Zandy Forbes. The piece explores Dr. Forbes’ multifaceted career, from academia and scientific research to finance to conceiving and founding the innovative gene therapy company. “This year, Forbes was named to Forbes Media’s 50 Over 50 class in the innovation category, and she said she was delighted to be recognized both as a woman scientist and as someone who didn’t have a ‘standard’ career path.”

Click to access the full profile.

Media & Honors: Forbes 50 > 50 Innovation List

MeiraGTx CEO and Founder Dr. Zandy Forbes has been honored with a spot on Forbes magazine’s 50 > 50: Innovation list for 2023, joining a trailblazing group of fellow leaders, entrepreneurs, and visionaries.

Click for the full feature.

Media: The Economist

In a recent piece on the business of gene therapies, The Economist cites MeiraGTx CEO and co-founder Zandy Forbes as an innovator focused on reversing rising costs in the field.

“[T]he company has decided to bring all its development and manufacturing in-house in order to radically reduce costs,” notes the article, in reference to MeiraGTx’s unique, end-to-end, vertically integrated approach to genetic medicine.

Click to view the full article. (Subscription log-in may be required.)

Media: BioWorld

“Ways to build better T cells.”

A recent piece by Mar de Miguel in BioWorld discusses the first “Spotlight on Immuno-Oncology” conference of the American Society of Gene & Cell Therapy (ASGCT) — with emphasis on MeiraGTx’s poster presentation marking the introduction of MeiraGTx’s CAR T technology, RiboCAR.

“MeiraGTx CAR T design is based on an orally available small molecule inducer that controls CAR expression to improve safety, efficacy, and durability,” writes de Miguel. “Their methodology resulted in naive T cells, which were less exhausted and more durable…. Unlike other modulable CARs that use chemicals for protein dimerization or viral protease, RiboCAR contains a synthetic Riboswitch ON created by Meiragtx, in which the aptamer acts as a sensor for a specific inducer. Without the inducer, CAR is undetectable and depends on an increasing dose, or it decreases after removing the small molecule inducer.”

The piece also cites extensive commentary from MeiraGTX CEO and Founder Zandy Forbes on the poster, its findings, and the significance of the innovation.

Click to access the full article (log-in may be required).

Media: BBC Click

“Exploring the Cutting-Edge Gene Therapy Helping Patients With Serious Diseases”

By Paul Carter
With MeiraGTx CEO Zandy Forbes
Video (4:51)

Media: National Public Radio (NPR) – Tech Nation with Moira Gunn

“Replacing Some Of Your DNA With New DNA That Works Better — and Stopping It At Will”

MeiraGTx CEO Zandy Forbes speaks with NPR’s Moira Gunn.

To learn about everything from our Phase 3 program in RPGR to our riboswitch gene regulation platform, listen to the interview.

Audio (59 mins)

Media:Bloomberg Surveillance
Hosted by Francine Lacqua

“MeiraGTx CEO on Potential of Gene Therapy, Innovation”

Interview
Video (4:31)

Manufacturing

MeiraGTx’s Industry-Leading Gene Therapy Manufacturing Facility in Shannon, Ireland Formally Introduced by Head of Irish Government, Micheál Martin

Public Event
Press Release

Conference: BofA Securities 2023 Health Care Conference

Fireside Chat with Alec Stranahan

Audio Replay (∼17 mins)

Clinical Pipeline: Salivary

MeiraGTx Announces Positive Clinical Data from the AQUAx Phase 1 Clinical Study of AAV2-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia

Clinical Program Update / Data Release

MeiraGTX at the European Society of Gene and Cell Therapy (ESGCT)

MeiraGTx Announces the Upcoming Presentation of 15 Abstracts at the European Society of Gene and Cell Therapy (ESGCT) 2022 Annual Congress.

Multiple Poster Presentations Highlight Versatility and Novelty of MeiraGTx’s Technology Platforms for Gene and Cell Therapy.

Press Release | Scientific Posters

Clinical Pipeline: Ocular

X-Linked Retinitis Pigmentosa (XLRP)

Late-Breaking Phase 1/2 Data Demonstrates Safety Profile of Investigational Gene Therapy Botaretigene Sparoparvovec (AAV-RPGR) and Sustained Vision Improvement in Patients with X-Linked Retinitis Pigmentosa (XLRP).

Results from the Phase 1/2 MGT009 study presented at the American Academy of Ophthalmology (AAO) 2022 Annual Meeting set the stage for Phase 3 LUMEOS trial of botaretigene sparoparvovec in XLRP, which is actively dosing patients.

Press Release | Data Release

Media: Sky News International – Ian King Live

“U.S. Clinical-Stage Gene Therapy Company MeiraGTX Has Opened The First Commercial-Scale Gene-Therapy Manufacturing Site in Ireland.”

Interview
Video (5 mins)

Media: Euronews Next

“This Gene Therapy Company is Testing New Tech to ‘Switch Off’ Diabetes and Obesity With a Pill”

By Natalie Huet
Article