Applied DNA Customer ÚHKT Initiates Phase I Clinical Trial for Rapidly Manufacturable CAR T-Cell Therapy Produced from Linea DNA
Applied DNA Sciences (NASDAQ:APDN) announced that the Czech Republic's State Institute for Drug Control approved a Phase I clinical trial for UHKT-CAR123-01, a CD123-specific autologous CAR T-cell therapy using Applied DNA's Linea DNA technology. The trial, conducted by the Institute of Hematology and Blood Transfusion (ÚHKT) in Prague, targets relapsed and/or refractory acute myeloid leukemia (AML).
The therapy aims to address high costs and long manufacturing times associated with traditional viral vector-based CAR T-cell therapies. Preclinical data demonstrated that ÚHKT's non-viral workflow using Linea DNA achieved rapid production of CAR T-cells with high potency while reducing manufacturing costs and timelines.
Applied DNA Sciences (NASDAQ:APDN) ha annunciato che l'Istituto Statale per il Controllo dei Farmaci della Repubblica Ceca ha approvato uno studio clinico di Fase I per UHKT-CAR123-01, una terapia CAR T-cell autologa specifica per CD123 che utilizza la tecnologia Linea DNA di Applied DNA. Lo studio, condotto dall'Istituto di Ematologia e Trasfusione del Sangue (ÚHKT) di Praga, si concentra sulla leucemia mieloide acuta (AML) in recidiva e/o refrattaria.
La terapia mira a affrontare gli elevati costi e i lunghi tempi di produzione associati alle terapie CAR T-cell tradizionali basate su vettori virali. I dati preclinici hanno dimostrato che il flusso di lavoro non virale di ÚHKT utilizzando Linea DNA ha raggiunto una produzione rapida di CAR T-cell con elevata potenza, riducendo al contempo i costi e i tempi di produzione.
Applied DNA Sciences (NASDAQ:APDN) anunció que el Instituto Estatal de Control de Medicamentos de la República Checa aprobó un ensayo clínico de Fase I para UHKT-CAR123-01, una terapia CAR T-cell autóloga específica para CD123 que utiliza la tecnología Linea DNA de Applied DNA. El ensayo, realizado por el Instituto de Hematología y Transfusión de Sangre (ÚHKT) en Praga, está dirigido a la leucemia mieloide aguda (AML) en recaída y/o refractaria.
La terapia tiene como objetivo abordar los altos costos y los largos tiempos de fabricación asociados con las terapias CAR T-cell tradicionales basadas en vectores virales. Los datos preclínicos demostraron que el flujo de trabajo no viral de ÚHKT utilizando Linea DNA logró una producción rápida de CAR T-cells con alta potencia, al tiempo que redujo los costos y los tiempos de fabricación.
Applied DNA Sciences (NASDAQ:APDN)은 체코 공화국의 국립 의약품 통제 기관이 Applied DNA의 Linea DNA 기술을 사용하는 CD123 특이적 자가 CAR T세포 요법인 UHKT-CAR123-01의 1상 임상 시험을 승인했다고 발표했습니다. 프라하에 위치한 혈액학 및 혈액 수혈 연구소(ÚHKT)에서 진행되는 이 시험은 재발성 또는 불응성 급성 골수성 백혈병(AML)을 목표로 합니다.
이 요법은 기존의 바이러스 벡터 기반 CAR T세포 요법과 관련된 높은 비용과 긴 제조 시간을 해결하는 것을 목표로 합니다. Preclinical 데이터는 Linea DNA를 사용하는 ÚHKT의 비바이러스 작업 흐름이 높은 효능을 가진 CAR T세포의 신속한 생산을 달성하면서 제조 비용과 시간을 줄였음을 보여주었습니다.
Applied DNA Sciences (NASDAQ:APDN) a annoncé que l'Institut d'État pour le contrôle des médicaments de la République tchèque a approuvé un essai clinique de phase I pour UHKT-CAR123-01, une thérapie CAR T-cell autologue spécifique à CD123 utilisant la technologie Linea DNA d'Applied DNA. L'essai, mené par l'Institut d'Hématologie et de Transfusion Sanguine (ÚHKT) à Prague, cible les cas de leucémie myéloïde aiguë (LMA) en rechute et/ou réfractaires.
La thérapie vise à remédier aux coûts élevés et aux longs délais de fabrication associés aux thérapies CAR T-cell traditionnelles basées sur des vecteurs viraux. Les données précliniques ont démontré que le flux de travail non viral de l'ÚHKT utilisant Linea DNA a permis une production rapide de CAR T-cells avec une forte puissance tout en réduisant les coûts et les délais de fabrication.
Applied DNA Sciences (NASDAQ:APDN) gab bekannt, dass das Staatsinstitut für Arzneimittelkontrolle der Tschechischen Republik eine Phase-I-Studie für UHKT-CAR123-01 genehmigt hat, eine CD123-spezifische autologe CAR-T-Zelltherapie, die die Linea-DNA-Technologie von Applied DNA nutzt. Die Studie, die vom Institut für Hämatologie und Bluttransfusion (ÚHKT) in Prag durchgeführt wird, zielt auf die rezidivierende und/oder refraktäre akute myeloische Leukämie (AML) ab.
Die Therapie zielt darauf ab, die hohen Kosten und langen Produktionszeiten zu adressieren, die mit traditionellen, auf viralen Vektoren basierenden CAR-T-Zelltherapien verbunden sind. Präklinische Daten zeigten, dass der nicht-virale Arbeitsablauf von ÚHKT unter Verwendung von Linea DNA eine schnelle Produktion von CAR-T-Zellen mit hoher Potenz erreichte und gleichzeitig die Produktionskosten und -zeiten senkte.
- Regulatory approval received for Phase I clinical trial using company's Linea DNA technology
- Preclinical data showed rapid and cost-efficient CAR T-cell production with high potency
- Additional customer clinical trials expected in calendar 2025
- None.
Insights
This clinical trial approval marks a significant advancement in CAR T-cell therapy manufacturing. The use of Linea DNA technology addresses two critical barriers in CAR-T development: high production costs and lengthy manufacturing times. Preclinical data demonstrates that the non-viral workflow produces high-yield, potent CAR T-cells more efficiently than traditional viral vector methods.
The Phase I trial targeting CD123 in relapsed/refractory AML represents a important development for patients with treatment options. The potential for faster, more cost-effective manufacturing could significantly improve accessibility to CAR-T therapies. This regulatory approval validates Linea DNA's potential as a disruptive technology in the cell therapy manufacturing space.
This regulatory milestone strengthens Applied DNA's position in the rapidly growing cell therapy manufacturing market. The company's Linea DNA platform could capture significant market share by addressing the cost and time inefficiencies that currently plague CAR-T production. With additional customers expected to initiate clinical trials in 2025, this successful regulatory approval could accelerate adoption of the technology and drive revenue growth.
For a company with a market cap of just
- Regulatory Approval Showcases Clinical Advancement of Linea DNA™ as a Rapid and Effective Means of Producing CAR-T cell Therapies -
STONY BROOK, NY / ACCESSWIRE / December 18, 2024 / Applied DNA Sciences, Inc. (NASDAQ:APDN) ("Applied DNA" or the "Company"), a leader in PCR-based DNA technologies, announced that the State Institute for Drug Control of the Czech Republic (SÚKL) approved an application for a Phase I clinical trial of an investigational CD123-specific autologous CAR T-cell therapy by the Institute of Hematology and Blood Transfusion (ÚHKT/Eng: IHBT) in Prague for the treatment of relapsed and/or refractory acute myeloid leukemia (AML). UHKT-CAR123-01 utilizes Applied DNA's synthetic DNA, Linea™ DNA, as a critical component in its manufacture.
AML is a hematologic malignancy with a high rate of treatment failure for which current treatment options are often restricted to palliative approaches. Novel emerging methods leveraging advancements in genetic medicines, such as CAR T-cell therapies, can potentially improve outcomes of patients after relapse but have been difficult to establish for clinical use due to high costs and long manufacturing times predominantly attributable to the use of viral vectors.
UHKT-CAR123 seeks to address these issues by generating CD123-specific CAR T-cells in a non-viral workflow utilizing Linea DNA to reduce manufacturing costs and timelines. Preclinical data showed that ÚHKT's Linea DNA-empowered non-viral workflow resulted in the rapid production of substantial and cost-efficient CAR T-cell yields with high potency[1].
"The use of Linea DNA illustrates our innovative approach to finding new and best-in-class treatments for patients with relapsed or refractory AML," stated Dr. Jan Vydra, principal investigator of the UHKT-CAR123 clinical trial.
Added Pavel Otáhal, Ph.D., scientific project leader at ÚHKT, "The Linea DNA platform enables the very rapid abiotic production of expression vectors usable for highly effective electroporation-based CAR-T manufacturing compared to plasmid-based vectors. The decreases in complexities and costs of developing autologous CAR-T technologies offer an innovative approach for the rapid clinical testing of novel types of CAR-T products. This is an incredible milestone for ÚHKT and one that we could not have achieved without the commitment of the Applied DNA team."
Applied DNA CEO Dr. James A. Hayward, said, "We congratulate ÚHKT on their progress into the clinic. Their accomplishment is also a significant milestone for Linea DNA as we look to support additional customers expected to initiate clinical trials in calendar 2025."
About Linea™ DNA
Linea™ DNA is an enzymatically produced, linear DNA manufactured by the Company's proprietary, large-scale polymerase chain reaction ("PCR") based manufacturing platform, the Linea™ DNA platform. As an alternative to plasmid-based DNA, Linea DNA can be used to manufacture of a wide range of nucleic acid-based therapies and in vitro diagnostics, including mRNA therapies, DNA vaccines, cell and gene therapies, and molecular and genetic diagnostic tests.
About Applied DNA Sciences
Applied DNA Sciences is a biotechnology company developing technologies to produce and detect deoxyribonucleic acid ("DNA"). Using the polymerase chain reaction ("PCR") to enable both the production and detection of DNA, we currently operate in three primary business markets: (i) the enzymatic manufacture of synthetic DNA for use in the production of nucleic acid-based therapeutics and the development and sale of a proprietary RNA polymerase ("RNAP") for use in the production of mRNA therapeutics; (ii) the detection of DNA and RNA in molecular diagnostics and genetic testing services; and (iii) the manufacture and detection of DNA for industrial supply chain security services.
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Forward-Looking Statements
The statements made by Applied DNA in this press release may be "forward-looking" in nature within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. Forward-looking statements describe Applied DNA's future plans, projections, strategies, and expectations, and are based on assumptions and involve a number of risks and uncertainties, many of which are beyond the control of Applied DNA. Actual results could differ materially from those projected due to its history of net losses, limited financial resources, the unknown future demand for its biotherapeutics and/or diagnostic products and services, the unknown amount of revenues and profits that will result from its Linea IVT and or Linea DNA platforms, the unknown outcome of the IHBT clinical trial, unforeseeable adverse events that could be encountered in the IHBT clinical trial, the fact that there has never been a commercial drug product utilizing the Linea DNA and/or the Linea IVT platform approved for therapeutic use, and various other factors detailed from time to time in Applied DNA's SEC reports and filings, including its Annual Report on Form 10-K filed on December 17, 2024, and other reports it files with the SEC, which are available at www.sec.gov. Applied DNA undertakes no obligation to update publicly any forward-looking statements to reflect new information, events, or circumstances after the date hereof or to reflect the occurrence of unanticipated events unless otherwise required by law.
Contacts:
Investor Relations contact: Sanjay M. Hurry, 917-733-5573, sanjay.hurry@adnas.com
Program contact: Brian Viscount, 631-240-8877, brian.viscount@adnas.com
Web: https://investors.adnas.com/
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[1] LINEAR DNA PLATFORM FOR THE NON-VIRAL POINT-OF-CARE PRODUCTION OF. . . by Pavel Otáhal. Copyright (C) 2024 MULTILEARNING Group Inc. https://library.ehaweb.org/eha/2024/eha2024-congress/419809/pavel.othal.linear.dna.platform.for.the.non-viral.point-of-care.production.of.html
SOURCE: Applied DNA Sciences, Inc.
View the original press release on accesswire.com
FAQ
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How does APDN's Linea DNA technology improve CAR T-cell therapy manufacturing?
What were the preclinical results for ÚHKT's CAR T-cell therapy using APDN's Linea DNA?
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