Design Therapeutics to Present Phase 1 Data for Fuchs Endothelial Corneal Dystrophy Program at Eyecelerator @ Park City 2025
Design Therapeutics (DSGN) announced it will present Phase 1 safety data for its DT-168 program at Eyecelerator @ Park City 2025 on May 2, 2025. The presentation will cover findings from single- and multiple-ascending dose trials in healthy adult volunteers and outline Phase 2 development plans for Fuchs endothelial corneal dystrophy (FECD) patients.
DT-168 is a GeneTAC® small molecule delivered as an eye drop, designed to target CTG repeat expansion in the TCF4 gene and reduce mutant gene product expression that causes corneal endothelial cell dysfunction in FECD. The company aims to develop the first disease-modifying treatment for FECD, a progressive corneal disease that currently often requires corneal transplant surgery.
Design Therapeutics (DSGN) ha annunciato che presenterà i dati di sicurezza di Fase 1 per il suo programma DT-168 all'Eyecelerator @ Park City 2025 il 2 maggio 2025. La presentazione includerà i risultati degli studi a dose singola e a dose multipla crescente su volontari adulti sani e illustrerà i piani di sviluppo della Fase 2 per i pazienti affetti da distrofia endoteliale corneale di Fuchs (FECD).
DT-168 è una piccola molecola GeneTAC® somministrata come collirio, progettata per colpire l'espansione delle ripetizioni CTG nel gene TCF4 e ridurre l'espressione del prodotto genico mutato che causa il malfunzionamento delle cellule endoteliali corneali nella FECD. L'azienda punta a sviluppare il primo trattamento che modifica la malattia per la FECD, una patologia corneale progressiva che spesso richiede un trapianto di cornea.
Design Therapeutics (DSGN) anunció que presentará datos de seguridad de la Fase 1 para su programa DT-168 en Eyecelerator @ Park City 2025 el 2 de mayo de 2025. La presentación incluirá los hallazgos de ensayos de dosis únicas y múltiples ascendentes en voluntarios adultos sanos, y describirá los planes de desarrollo de la Fase 2 para pacientes con distrofia endotelial corneal de Fuchs (FECD).
DT-168 es una pequeña molécula GeneTAC® administrada en forma de gotas oftálmicas, diseñada para dirigirse a la expansión de repeticiones CTG en el gen TCF4 y reducir la expresión del producto génico mutante que provoca la disfunción de las células endoteliales corneales en FECD. La compañía busca desarrollar el primer tratamiento modificador de la enfermedad para FECD, una enfermedad corneal progresiva que actualmente suele requerir cirugía de trasplante de córnea.
Design Therapeutics (DSGN)은 2025년 5월 2일 Eyecelerator @ Park City 2025에서 DT-168 프로그램의 1상 안전성 데이터를 발표할 예정입니다. 발표에서는 건강한 성인 지원자를 대상으로 한 단회 및 다회 점증 용량 시험 결과를 다루고, Fuchs 각막내피이영양증 (FECD) 환자를 위한 2상 개발 계획을 설명할 것입니다.
DT-168은 GeneTAC® 소분자로 안약 형태로 투여되며, TCF4 유전자의 CTG 반복 확장을 표적으로 하여 FECD에서 각막내피세포 기능장애를 일으키는 돌연변이 유전자 산물의 발현을 줄이도록 설계되었습니다. 이 회사는 현재 각막 이식 수술이 자주 필요한 진행성 각막 질환인 FECD에 대한 최초의 질병 변형 치료제를 개발하는 것을 목표로 하고 있습니다.
Design Therapeutics (DSGN) a annoncé qu'elle présenterait les données de sécurité de Phase 1 pour son programme DT-168 lors de l'Eyecelerator @ Park City 2025, le 2 mai 2025. La présentation couvrira les résultats des essais à dose unique et à doses multiples croissantes chez des volontaires adultes sains, et exposera les plans de développement de Phase 2 pour les patients atteints de dystrophie endothéliale de Fuchs (FECD).
DT-168 est une petite molécule GeneTAC® administrée sous forme de collyre, conçue pour cibler l'expansion des répétitions CTG dans le gène TCF4 et réduire l'expression du produit génétique mutant responsable du dysfonctionnement des cellules endothéliales cornéennes dans la FECD. La société vise à développer le premier traitement modifiant la maladie pour la FECD, une maladie cornéenne progressive qui nécessite souvent une greffe de cornée à l'heure actuelle.
Design Therapeutics (DSGN) gab bekannt, dass es am 2. Mai 2025 auf der Eyecelerator @ Park City 2025 Sicherheitsdaten der Phase 1 für sein DT-168-Programm vorstellen wird. Die Präsentation wird Ergebnisse aus Einzel- und mehrfach ansteigenden Dosisstudien bei gesunden erwachsenen Probanden enthalten und die Entwicklungspläne für Phase 2 bei Patienten mit Fuchs-Endothel-Dystrophie (FECD) skizzieren.
DT-168 ist ein als Augentropfen verabreichtes GeneTAC®-Kleinstmolekül, das darauf ausgelegt ist, die CTG-Repeat-Expansion im TCF4-Gen zu adressieren und die Expression des mutierten Genprodukts zu verringern, das die Funktionsstörung der Hornhautendothelzellen bei FECD verursacht. Das Unternehmen strebt die Entwicklung der ersten krankheitsmodifizierenden Behandlung für FECD an, eine fortschreitende Hornhauterkrankung, die derzeit häufig eine Hornhauttransplantation erfordert.
- Development of first potential disease-modifying treatment for FECD
- Convenient eye drop formulation for drug delivery
- Successful completion of Phase 1 trial leading to Phase 2 planning
- Early-stage development with no efficacy data presented yet
- No current revenue generation from product
Insights
Design Therapeutics will present Phase 1 safety data for its novel FECD eye drop therapy, advancing toward Phase 2 trials for an untreated genetic eye disease.
Design Therapeutics' upcoming presentation of Phase 1 safety findings for DT-168 represents a notable development milestone in their clinical program targeting Fuchs endothelial corneal dystrophy (FECD). The company will share data from both single and multiple-ascending dose trials in healthy volunteers at the Eyecelerator conference, alongside their plans for Phase 2 clinical development in actual FECD patients.
What makes DT-168 particularly interesting is its unique approach - it's formulated as an eye drop that selectively targets the CTG repeat expansion in the TCF4 gene responsible for causing corneal endothelial cell dysfunction in FECD. This GeneTAC small molecule is designed to reduce expression of the mutant gene product driving the disease progression.
The significance of this program stems from the current treatment landscape - FECD has no disease-modifying treatments available, with many patients ultimately requiring corneal transplant surgery. An eye drop that could potentially restore endothelial function would represent a major advancement in treatment paradigm.
For a clinical-stage biotech with Design's profile, the transition from Phase 1 to planned Phase 2 trials represents standard pipeline progression. While safety data is an important milestone, the true value-determining data will come from efficacy results in FECD patients in subsequent trials.
Phase 1 data for DT-168 eye drops targets genetic mutation causing FECD, potentially offering first non-surgical treatment option for this progressive corneal disease.
The upcoming presentation of Design Therapeutics' DT-168 Phase 1 data merits attention within the ophthalmology community for its potential to address an important unmet need. FECD is a progressive corneal disease that causes endothelial cell dysfunction, leading to corneal edema, visual impairment, and often culminating in the need for corneal transplantation.
DT-168's mechanism of action is particularly notable - it specifically targets the CTG repeat expansion in the TCF4 gene that underlies the pathology in many FECD patients. By reducing expression of the mutant gene product, it aims to address the genetic root cause rather than merely managing symptoms.
The eye drop formulation represents a significant potential advantage from both clinical and patient perspectives. Current FECD management is to symptomatic treatments (hypertonic saline drops for temporary relief) until disease progression necessitates corneal transplantation. A topical therapy that could potentially modify disease progression would dramatically change the treatment paradigm.
While the Phase 1 data being presented focuses on safety in healthy volunteers, the planned progression to Phase 2 studies in FECD patients signals confidence in the preliminary safety profile. The true test will come when efficacy is evaluated in actual patients - determining whether the genetic targeting mechanism translates to clinical improvements in corneal endothelial function and disease stabilization.
CARLSBAD, Calif., April 21, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that it will present an update on the progress of its DT-168 program for Fuchs endothelial corneal dystrophy (FECD) at Eyecelerator @ Park City 2025 on Friday, May 2, 2025, at 1:30 p.m. MT in Park City, UT. The presentation will include safety findings from the Phase 1 single- and multiple-ascending dose trial of DT-168 in healthy adult volunteers and plans for Phase 2 clinical development in FECD patients.
DT-168 is a GeneTAC® small molecule, formulated as an eye drop, that is designed to selectively target the CTG repeat expansion in the TCF4 gene and reduce the expression of the mutant gene product that causes corneal endothelial cell dysfunction leading to FECD.
“We are pleased for the opportunity to showcase our Phase 1 results for DT-168 at Eyecelerator, a forum dedicated to highlighting innovation in the field of ophthalmology,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “FECD is a progressive corneal disease with no disease-modifying treatments currently available, ultimately leading to corneal transplant surgery for many patients. We believe DT-168, formulated as an eye drop, has the potential to restore endothelial function, representing a significant advancement in the treatment of FECD.”
Eyecelerator conferences, backed by the American Academy of Ophthalmology, highlight industry advancements and innovative new products disrupting eye care. Eyecelerator @ Park City 2025 is adjacent to the Association for Research in Vision and Ophthalmology (ARVO) meeting being held May 4 - 8, 2025, in Salt Lake City, UT.
About Design Therapeutics
Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC® gene targeted chimera small molecules. The company’s GeneTAC® molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. In addition to its GeneTAC® programs, DT-216P2, in development for patients with Friedreich ataxia, and DT-168, for Fuchs endothelial corneal dystrophy, the company is advancing programs in myotonic dystrophy type-1 and Huntington’s disease. Discovery efforts are underway for multiple genomic medicines. For more information, please visit designtx.com.
Forward-Looking Statements
Statements in this press release that are not purely historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to the potential for DT-168 to restore endothelial function and its impact on the treatment of FECD; Design's ability to advance its pipeline of GeneTAC® small molecules; and the capabilities and potential advantages of Design’s pipeline of GeneTAC® small molecules. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “designed to,” “anticipates,” “capable of,” “plans to,” “expects,” “estimate,” “intends,” “will,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Design’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with: the acceptance of INDs by the FDA or similar applications by foreign regulatory agencies for the conduct of planned clinical trials of our product candidates and our proposed design of future clinical trials; nonclinical development activities and results of nonclinical studies; conducting a clinical trial and patient enrollment, which are affected by many factors, and any difficulties or delays encountered with such clinical trial or patient enrollment may delay or otherwise adversely affect Design’s clinical development plans; the process of discovering and developing therapies that are safe and effective for use as human therapeutics and operating as a development stage company; undesirable side effects or other undesirable properties, which could cause Design or regulatory authorities to suspend or discontinue clinical trials and thereby delay or prevent Design’s product candidates’ development or regulatory approval; Design’s ability to develop, initiate or complete nonclinical studies and clinical trials for its product candidates; whether promising early research or clinical trials will demonstrate safety and/or efficacy in later nonclinical studies or clinical trials; changes in Design’s plans to develop its product candidates; reliance on third parties to successfully conduct clinical trials and nonclinical studies; competitive products, which may make any products we develop or seek to develop obsolete or noncompetitive; Design’s reliance on key third parties, including contract manufacturers and contract research organizations; Design’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; regulatory developments in the United States and foreign countries; Design’s ability to obtain and maintain intellectual property protection for its product candidates; Design’s ability to recruit and retain key scientific or management personnel; and market conditions. For a more detailed discussion of these and other factors, please refer to Design’s filings with the Securities and Exchange Commission (SEC), including under the “Risk Factors” heading of Design’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024, as filed with the SEC on March 10, 2025. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Design undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.
Contact:
Renee Leck
THRUST Strategic Communications
renee@thrustsc.com
FAQ
What are the key findings from Design Therapeutics' (DSGN) Phase 1 trial of DT-168?
How does Design Therapeutics' DT-168 treatment work for FECD patients?
When will Design Therapeutics (DSGN) begin Phase 2 trials for DT-168?
What is the current treatment landscape for Fuchs endothelial corneal dystrophy (FECD)?
