Envisioning a Future with
Next-Gen Genetic Medicine

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. 

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. 

Click through for the full article.

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.) 

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 recently announced positive data from our clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease. An article from Parkinson’s News Today — “AAV-GAD Gene Therapy Eases Parkinson’s Symptoms in Early Study” — provides a good summary of the findings and potential impact.

“Treatment with the experimental gene therapy AAV-GAD led to significant easing of motor symptoms and improved life quality for most people with Parkinson’s disease in an early clinical trial, according to new data announced by the therapy’s developer MeiraGTx.”

Click through for the full article.

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