MeiraGTx

Zandy Forbes, CEO and co-founder of MeiraGTx, will appear today, June 12, at the Goldman Sachs 45th Annual Global Healthcare Conference. Dr. Forbes will discuss the latest innovations in genetic medicine from MeiraGTx, the company's end-to-end manufacturing and business models, and more.    Tune in live to Dr. Forbes's comments at 2:40pm EST U.S. time today here: https://lnkd.in/enH38PS4

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease for which there is currently no cure. MeiraGTx is developing a first-in-class gene therapy candidate to potentially address both familial and sporadic ALS (>95% of ALS patients). Data resulting from MeiraGTx’s in vivo preclinical trials held in collaboration with leading ALS researchers provide strong evidence for the therapeutic potential of MeiraGTx’s genetic medicine (known as AAV-hUPF1). At the ALS Drug Development Summit in Boston today, Dr. Barbara Nguyen-Vu, Senior Director of Neuro at MeiraGTx, will discuss these data and insights in her presentation, “Demonstrating Success of a One-Dose Gene Therapy to Prevent Motor Neuronal Loss in Both Sporadic & Familial ALS: Preclinical Efficacy of AAV-hUPF1 Optimized for Clinical Translation with Improved Vector Genome and Novel CNS Capsid.” https://lnkd.in/gCHds7tt

A fantastic piece of coverage in The Sunday Times featuring our client MeiraGTx's chief executive, Zandy Forbes. In a fascinating interview with Jim Armitage, Zandy talks about her life, her three careers and MeiraGTx's innovative business model. Read the full piece, here: https://lnkd.in/e54WqyfS

ICYMI: MeiraGTx’s gene therapy 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 of the gene therapy is now enrolling eligible participants. Find more information and the full presentation here: https://lnkd.in/epEPuFmC

Our CEO, Zandy Forbes, recently sat down with Brad Loncar at the NYSE for a segment of BiotechTV. Watch the full episode for more on our latest pipeline progress as well as our differentiated #GeneTherapy manufacturing capabilities.

Today we announced Q4 and full year 2023 financial and operational results. Learn more about the progress we've made across every area of the company: https://brnw.ch/21wHRR4.

Join us in sharing your colors for #RareDiseaseDay! Today is an opportunity to build awareness of #RareDiseases that affect millions of patients worldwide. Together, let's illuminate the path toward a future where rare diseases are understood, acknowledged, and met with solutions.

Many chronic and inherited retinal diseases (#IRDs) are progressive, resulting in many patients experiencing blindness. For #LowVisionAwarenessMonth, we're spreading awareness of #AMD and other diseases that cause visual impairment. Learn more here: https://brnw.ch/21wH0qU

In case you missed it, we recently entered into an asset purchase agreement with Johnson & Johnson Innovative Medicine for the remaining interests in our potential #GeneTherapy treatment for X-linked retinitis pigmentosa (XLRP). Learn more about this transaction worth up to $415 million here: https://lnkd.in/eerDn-Px

In October, BioSpace's Nadia Bey profiled the multifaceted and inspiring career journey of our CEO Zandy Forbes, from her days in academic genetics research to biotech investing to the founding and growth of MeiraGTx. https://lnkd.in/eFa-K_ew