MeiraGTx Announces Two Posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress
MeiraGTx (Nasdaq: MGTX) will present two posters at the ESGCT 2025 Annual Congress in Seville, Oct 7-10, 2025.
Poster P0089 reports directed evolution of novel AAV capsids from a ~1E7-variant library in non-human primate eyes, validated in human retinal organoids and iPSC-derived RPE. Selected capsids showed >2-fold higher transduction versus AAV2.7m8 and higher retinal expression in mouse intravitreal studies, supporting ocular gene therapy applications.
Poster P0170 describes scale-up of a perfusion-based AAV manufacturing process to 40L bioreactors. Bench optimization delivered up to +120% volumetric VG yield, ~50% lower plasmid use, and up to 2.2-fold lower cost-of-goods per dose; the work focuses on translating these parameters to 40L while preserving quality.
MeiraGTx (Nasdaq: MGTX) presenterà due poster al ESGCT 2025 Annual Congress a Siviglia, dal 7 al 10 ottobre 2025.
Poster P0089 riporta l'evoluzione diretta di nuovi capside AAV da una libreria di ~1E7 varianti in occhi di primati non umani, con validazione in organoidi retinali umani e RPE derivata da iPSC. I capside selezionati hanno mostrato una trasduzione >2 volte superiore rispetto ad AAV2.7m8 e una maggiore espressione retinica negli studi intravitreali su topo, supportando applicazioni di terapia genica oculare.
Poster P0170 descrive la scalatura di un processo di produzione AAV basato su perfusione a bioreattori da 40 L. L'ottimizzazione in laboratorio ha fornito fino a un aumento di +120% del rendimento volumetrico di VG, circa il 50% in meno di utilizzo di plasmide e fino a 2,2 volte in meno costo-per-dose; il lavoro si concentra sul trasferimento di questi parametri a 40 L mantenendo la qualità.
MeiraGTx (Nasdaq: MGTX) presentará dos pósters en el ESGCT 2025 Annual Congress en Sevilla, del 7 al 10 de octubre de 2025.
Póster P0089 reporta la evolución dirigida de nuevas cápsides AAV a partir de una biblioteca de ~1E7 variantes en ojos de primates no humanos, validada en organoides retinales humanos y RPE derivada de iPSC. Las cápsides seleccionadas mostraron una transducción >2 veces mayor frente a AAV2.7m8 y una mayor expresión retiniana en estudios intravítreos en ratón, respaldando aplicaciones de terapia génica ocular.
Póster P0170 describe la escalada de un proceso de fabricación de AAV basado en perfusión a biorreactores de 40 L. La optimización de banco proporcionó hasta +120% de rendimiento volumétrico de VG, ~50% menos uso de plásmido y hasta 2,2 veces menos costo de bienes por dosis; el trabajo se centra en trasladar estos parámetros a 40 L manteniendo la calidad.
MeiraGTx (Nasdaq: MGTX)는 2025년 ESGCT 연례 학술대회에서 두 편의 포스터를 세비야에서 발표합니다. 기간은 2025년 10월 7일부터 10일까지.
포스터 P0089는 비인간 영장류 눈에서 ~1E7 변이 라이브러리로부터 생성된 새로운 AAV 캡시드의 지향적 진화를 보고하며, 인간 망막 오가노이드 및 iPSC 유래 RPE에서 검증되었습니다. 선택된 캡시드는 AAV2.7m8에 비해 트랜스덕션이 2배 이상 높았고, 생쥐의 안구내 주사 연구에서 망막 발현이 더 높아 눈 치료용 유전자 치료 응용 가능성을 뒷받침합니다.
포스터 P0170은 40L 바이오리액터로의 순환 기반 AAV 제조 프로세스 확장을 설명합니다. 벤치 최적화로 VG 용량 수율이 최대 +120%, 플라스미드 사용량 약 50% 감소, 단가당 비용이 최대 2.2배 낮아졌으며; 이 연구는 품질을 유지하면서 40L로 이러한 매개변수를 이전하는 데 중점을 둡니다.
MeiraGTx (Nasdaq: MGTX) présentera deux posters au ESGCT 2025 Annual Congress à Séville, du 7 au 10 octobre 2025.
Poster P0089 rapporte une évolution dirigée de nouveaux capsides AAV à partir d’une bibliothèque d’environ 1E7 variantes chez des yeux de primates non humains, validée dans des organoïdes rétiniens humains et une RPE dérivée d’iPSC. Les capsides sélectionnées ont montré une transduction >2 fois supérieure à AAV2.7m8 et une expression rétinienne plus élevée dans des études intravitréennes chez la souris, soutenant les applications de thérapie génique oculaire.
Poster P0170 décrit l’échelle d’un procédé de fabrication d’AAV basé sur la perfusion jusqu’à des réacteurs de 40 L. L’optimisation en banc a permis jusqu’à +120% de rendement volumétrique VG, environ 50% de réduction de l’utilisation de plasmide, et jusqu’à 2,2 fois de coût des biens par dose; le travail se concentre sur la traduction de ces paramètres à 40 L tout en préservant la qualité.
MeiraGTx (Nasdaq: MGTX) wird auf dem ESGCT 2025 Annual Congress in Sevilla zwei Poster präsentieren, vom 7. bis 10. Oktober 2025.
Poster P0089 berichtet über die gerichtete Evolution neuartiger AAV-Kapside aus einer Bibliothek von ca. 1E7 Varianten in Augen von Nicht-Mensch-Primaten, validiert in menschlichen Retina-Organoiden und iPSC-abgeleiteter RPE. Ausgewählte Kapside zeigten eine >2-fach höhere Transduktion im Vergleich zu AAV2.7m8 und eine höhere retinalen Expression in Maus-Intravitrealis-Studien, was ocularen Gentherapie-Anwendungen zugutekommt.
Poster P0170 beschreibt die Hochskalierung eines perfusionsbasierten AAV-Herstellungsprozesses auf 40-L-Bioreaktoren. Bench-Optimierung führte zu bis zu +120% volumetrische VG-Ausbeute, etwa 50% geringerem Plasmidverbrauch und bis zu 2,2-fach niedrigerem Kostensatz pro Dosis; die Arbeit konzentriert sich darauf, diese Parameter auf 40 L zu übertragen und dabei die Qualität zu erhalten.
MeiraGTx (المدرجة في ناسداك: MGTX) ستقدم wandelen عناوين لوحة في ESGCT 2025 Annual Congress في إشبيلية، من 7 إلى 10 أكتوبر 2025.
اللوحة P0089 تقارير التطوير الموجّه لمكونات AAV الجديدة من مكتبة تحتوي على حوالي 1E7 متغيرًا في عيون رخويات غير بشرية، وقد تم التحقق منها في عضيات الشبكية البشرية وRPE مشتقة من iPSC. أظهرت الأسطح المحددة معدل ترميز أعلى من AAV2.7m8 وتعبيرًا شبكياً أعلى في دراسات داخل العين على فئران، مما يدعم تطبيقات العلاج الجيني العيني.
اللوحة P0170 تصف توسيع عملية تصنيع AAV المعتمدة على التروية إلى مفاعلات حيوية بسعة 40 لترًا. التحسين التجريبي قدّم حتى +120% من عائد VG حجمي، حوالي 50% تقليلًا في استخدام البلازميد، وحتى انخفاض بقيمة 2.2 مرة في تكلفة السلع لكل جرعة؛ يعمل المشروع على ترحيل هذه المتغيرات إلى 40 لتر مع الحفاظ على الجودة.
MeiraGTx (纳斯达克股票代码：MGTX) 将在 ESGCT 2025 年年会于塞维利亚于 2025 年 10 月 7-10 日发布两份海报。
海报 P0089 报告了来自 ~1E7 种变体库的新的 AAV 衬层的定向进化，在非人灵长类动物眼中得到验证，并在人类视网膜类器官及 iPSC 来源的 RPE 中得到验证。所选衬层在对比 AAV2.7m8 的转导效率上提高了>2 倍，并且在小鼠玻璃体内研究中对视网膜的表达也更高，支持眼科基因治疗的应用。
海报 P0170 描述了将基于灌流的 AAV 生产工艺扩大到 40 L 生物反应器的过程。基准优化实现了 VG 体积产率最高提升 +120%，质粒使用量降低约 50%，单位剂成本降低至最高 2.2 倍；该工作专注于在保持质量的前提下，将这些参数转化到 40 L 条件。
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Multiple Posters Highlight the Breadth of Company’s Novel Genetic Medicine and Cell Therapy Platforms
LONDON and NEW YORK, Oct. 07, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the Company will exhibit two posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, which is being held from October 7-10, 2025, in Seville, Spain.
The posters are available on the Posters and Publications page of the Company’s website.
The details of the poster presentations are as follows:
Poster Number: P0089
Abstract Title: Novel AAV Capsids for Intravitreal Delivery Developed by Directed Evolution in Non-human Primate Eyes and Validated in Human Retinal Organoids
Poster Session: Wednesday 8 October from 14:00 to 15:30 CEST
Abstract:
We performed an in vivo directed evolution capsid screen in non-human primates to identify novel capsids that target the back of the eye. This involved administering a diverse library of AAV capsid variants intravitreally into the eyes of NHPs.
The library was constructed by generating a variety of AAV capsid variants through the insertion of random peptides at a specific site in the capsid. Specifically, a 7-mer peptide display library based on AAV2 was created, with a 7-mer peptide inserted at amino acid 588, flanked by a 5' AAA linker and a 3' AA linker.
After administration, the fovea was excised, and retinal cells were harvested. By excluding the fovea, which most capsids readily transduce, we aimed to identify capsids with a broader transduction profile, extending to the perimacular region and beyond. Viral genomes were recovered and sequenced to identify the most efficient variants. By analysing the recovered viral genomes, we identified the top-performing capsids for further development.
From a library of ~ 1E7 variants, the top-performing capsids were selected. These candidates were validated and compared head-to-head against AAV2.7m8 and the parental AAV2 in our human retinal organoid platform using live cell imaging, flow cytometry, histology, and single-cell RNA sequencing. We identified variants with significantly higher transduction efficiency compared to the parental serotype, AAV2, and AAV2.7m8. Deep transcriptional profiling using single-cell RNA sequencing demonstrated over 2-fold higher transduction efficiency of our novel capsids compared to AAV2.7m8. Top performers were further validated and characterized in human iPSC-derive RPE where our novel capsids showed higher transduction efficiency compared to other natural and engineered serotypes. In addition, in vivo studies in mice using absolute quantification of total retinal expression in intravitreally injected retinas, overall expression levels of our novel capsids were higher than AAV2.7m8.
These novel AAV capsids have potential applications in treating various ocular diseases, including inherited retinal disorders and age-related macular degeneration. Their enhanced efficiency makes them exceptional candidates for gene therapy approaches requiring precise targeting of retinal cells and high overall levels of retinal transduction and expression. The ability to transduce a broad range of retinal cell types with high efficiency opens up new possibilities for treating complex retinal diseases that affect multiple cell types in the retina.
Poster Number: P0170
Abstract Title: Scale-up of a Perfusion-Based AAV Manufacturing Process
Poster Session: Thursday 9 October from 14:00 to 15:30 CEST
Abstract:
Building upon prior development of MeiraGTx’s adeno-associated viral vector (AAV) upstream manufacturing platform, this work presents the scale-up of our perfusion-based AAV production process to 40L bioreactors. MeiraGTx’s perfusion-based upstream process was optimized and modulated through the choice of transfection reagents, AAV production enhancers, and transfection parameters, in 250mL stirred tank reactors. Operating a perfusion-based process at bench-scale was found to increase volumetric VG yield by up to
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline with four late-stage clinical programs. Each of these programs use local delivery of small doses resulting in disease modifying effects in both inherited and more common diseases, in the eye, Parkinson’s disease and radiation-induced xerostomia. MeiraGTx uses its innovative technology in optimization of capsids, promoters and novel translational control elements to develop best in class, potent, safe viral vectors. MeiraGTx’s broad pipeline is supported by end-to-end in-house manufacturing. MeiraGTx has built the most comprehensive manufacturing capabilities in the industry, with 5 facilities globally, including two that are licensed for GMP viral vector production and a GMP QC facility with clinical and commercial licensure. In addition, MeiraGTx has developed a proprietary manufacturing platform process over 9 years based on more than 20 different viral vectors with leading yield and quality aspects and commercial readiness. Uniquely, MeiraGTx has developed a novel technology for in vivo delivery of any biologic therapeutic using oral small molecules. This transformative riboswitch gene regulation technology allows precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the regulated in vivo delivery of metabolic peptides, including GLP-1, GIP, Glucagon, Amylin, PYY and Leptin, as well as cell therapy, CAR-T for liquid and solid tumors and autoimmune diseases, and additionally PNS targets addressing long term intractable pain. MeiraGTx has developed the technology to apply genetic medicine to common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.
For more information, please visit www.meiragtx.com
Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our product candidate development and anticipated milestones regarding our pre-clinical and clinical data, reporting of such data and the timing of results of data and regulatory matters, , as well as statements that include the words “expect,” “will,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “could,” “should,” “would,” “continue,” “anticipate” and similar statements of a future or forward-looking nature. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, repay our debt obligations, identify additional and develop existing product candidates, successfully execute strategic transactions or priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug or rare pediatric disease designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of pandemics, epidemics or outbreaks of infectious diseases on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward- looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.
Contacts
Investors:
MeiraGTx
Investors@meiragtx.com
or
Media:
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com
FAQ
What did MeiraGTx announce at ESGCT 2025 about new AAV capsids (MGTX)?
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