Generation Bio to Apply ctLNP Delivery Technology to Develop siRNA Therapeutics for T Cell-Driven Autoimmune Diseases
Generation Bio (NASDAQ:GBIO) announced its strategic focus on developing siRNA therapeutics for T cell-driven autoimmune diseases using their cell-targeted lipid nanoparticle (ctLNP) delivery technology. The company aims to selectively modulate T cells in vivo by silencing hard-to-drug targets.
Recent non-human primate data showed ctLNPs effectively target T cells with strong selectivity for CD8+ and CD4+ effector T cells and NK cells. The company plans to submit its first IND in the second half of 2026 and enter clinical trials within its cash runway, which extends into the second half of 2027.
The company announced organizational changes, including Phillip Samayoa, Ph.D. succeeding Matthew Stanton, Ph.D. as chief scientific officer, and Kevin Conway being elected as chief financial officer. A chief medical officer appointment is expected in 2025.
Generation Bio (NASDAQ:GBIO) ha annunciato il suo focus strategico nello sviluppo di terapeutiche siRNA per malattie autoimmuni guidate da cellule T, utilizzando la loro tecnologia di consegna a nanoparticelle lipidiche mirate alle cellule (ctLNP). L'azienda mira a modulare selettivamente le cellule T in vivo silenziando bersagli difficili da trattare.
Dati recenti su primati non umani hanno mostrato che le ctLNP mirano efficacemente alle cellule T con una forte selettività per le cellule T effettrici CD8+ e CD4+ e le cellule NK. L'azienda prevede di presentare la sua prima richiesta IND nella seconda metà del 2026 e di entrare negli studi clinici entro il periodo di liquidità, che si estende nella seconda metà del 2027.
L'azienda ha annunciato cambiamenti organizzativi, tra cui Phillip Samayoa, Ph.D., che succede a Matthew Stanton, Ph.D., come direttore scientifico, e Kevin Conway eletto come direttore finanziario. È prevista un'assegnazione di un direttore medico nel 2025.
Generation Bio (NASDAQ:GBIO) anunció su enfoque estratégico en el desarrollo de terapias con siRNA para enfermedades autoinmunes impulsadas por células T, utilizando su tecnología de entrega de nanopartículas lipídicas dirigidas a células (ctLNP). La compañía tiene como objetivo modular selectivamente las células T in vivo al silenciar dianas difíciles de tratar.
Datos recientes de primates no humanos mostraron que las ctLNPs apuntan eficazmente a las células T con una fuerte selectividad por las células T efectoras CD8+ y CD4+ y las células NK. La empresa planea presentar su primera solicitud IND en la segunda mitad de 2026 e iniciar ensayos clínicos dentro de su período de liquidez, que se extiende hasta la segunda mitad de 2027.
La empresa anunció cambios organizativos, incluido Phillip Samayoa, Ph.D. que sucederá a Matthew Stanton, Ph.D. como director científico, y Kevin Conway elegido como director financiero. Se espera un nombramiento de un director médico en 2025.
Generation Bio (NASDAQ:GBIO)는 세포 T 주도의 자가면역 질환을 위한 siRNA 치료제 개발에 대한 전략적 초점을 발표하였으며, 이를 위해 세포 표적 지질 나노입자(ctLNP) 전달 기술을 사용합니다. 이 회사는 치료하기 어려운 타겟을 침묵시켜 vivo에서 세포 T를 선택적으로 조절하는 것을 목표로 하고 있습니다.
최근 비인간 영장류 데이터에 따르면 ctLNP는 CD8+ 및 CD4+ 효과 T 세포와 NK 세포에 대해 강한 선택성으로 T 세포를 효과적으로 표적화합니다. 회사는 2026년 하반기에 첫 IND를 제출하고 2027년 하반기까지 연장되는 자금 소진 내에서 임상 시험에 들어갈 계획입니다.
회사는 Phillip Samayoa, Ph.D.가 Matthew Stanton, Ph.D.를 CEO로서 후임하고, Kevin Conway가 CFO로 선출되었다고 발표했습니다. 2025년에는 CMO 임명이 예상됩니다.
Generation Bio (NASDAQ:GBIO) a annoncé son orientation stratégique vers le développement de thérapies siRNA pour les maladies auto-immunes dirigées par les cellules T, utilisant sa technologie de livraison de nanoparticules lipidiques ciblées (ctLNP). L'entreprise vise à moduler sélectivement les cellules T in vivo en silence des cibles difficiles à traiter.
Des données récentes sur des primates non humains ont montré que les ctLNP ciblent efficacement les cellules T avec une forte sélectivité pour les cellules T effectrices CD8+ et CD4+ ainsi que pour les cellules NK. L'entreprise prévoit de soumettre sa première demande IND dans la seconde moitié de 2026 et de commencer des essais cliniques dans la durée de ses liquidités, qui s'étend jusqu'à la seconde moitié de 2027.
L'entreprise a annoncé des changements organisationnels, y compris Phillip Samayoa, Ph.D. succédant à Matthew Stanton, Ph.D. en tant que directeur scientifique, et Kevin Conway élu directeur financier. La nomination d'un directeur médical est attendue en 2025.
Generation Bio (NASDAQ:GBIO) hat ihren strategischen Fokus auf die Entwicklung von siRNA-Therapeutika für T-Zell-gesteuerte Autoimmunerkrankungen mithilfe ihrer zielgerichteten Lipid-Nanopartikel (ctLNP) Verabreichungstechnologie angekündigt. Das Unternehmen zielt darauf ab, in vivo selektiv T-Zellen durch das Ausschalten schwer behandelbarer Ziele zu modulieren.
Aktuelle Daten von nichtmenschlichen Primaten zeigten, dass ctLNPs T-Zellen mit starker Selektivität gegenüber CD8+ und CD4+ Effektor-T-Zellen sowie NK-Zellen effektiv anvisieren. Das Unternehmen plant, seine erste IND in der zweiten Hälfte von 2026 einzureichen und im Rahmen der bis zur zweiten Hälfte von 2027 verlängerten Finanzmittel klinische Studien zu starten.
Das Unternehmen gab organisatorische Änderungen bekannt, darunter die Ernennung von Phillip Samayoa, Ph.D., als wissenschaftlichen Leiter, der Matthew Stanton, Ph.D., nachfolgt, sowie die Wahl von Kevin Conway als Finanzvorstand. Eine Ernennung eines medizinischen Leiters wird für 2025 erwartet.
- Validated ctLNP technology demonstrates selective targeting of T cells in non-human primate studies
- Cash runway extends into second half of 2027, covering planned clinical trial initiation
- Clear timeline for first IND submission in H2 2026
- No immediate revenue generation as clinical trials won't start until 2026
- Significant organizational restructuring may cause temporary operational disruption
- Early-stage technology with no human clinical data yet
Insights
Generation Bio's strategic pivot towards siRNA therapeutics using their ctLNP delivery platform represents a significant technological advancement in targeting T cell-driven autoimmune diseases. The company's non-human primate data demonstrating selective targeting of CD8+ and CD4+ effector T cells and NK cells validates their approach to a historically challenging delivery problem.
The technology's potential to selectively modulate T cell function while sparing broader immune system impact could provide a meaningful competitive advantage in the
The leadership reorganization, particularly the promotion of internal candidates, suggests confidence in the platform's direction while maintaining institutional knowledge. However, investors should note that the departure of key scientific leadership during this critical development phase requires careful monitoring.
The technical approach of using ctLNPs to deliver siRNA specifically to T cells addresses a important gap in autoimmune disease treatment. Current biologics often broadly suppress immune responses, leading to increased infection risks. The selective targeting of CD8+ and CD4+ effector T cells could enable more precise therapeutic intervention in conditions like rheumatoid arthritis, multiple sclerosis and type 1 diabetes.
The platform's ability to potentially modulate "undruggable targets" involved in T cell activation, differentiation and tissue migration could open new therapeutic possibilities. However, the success will heavily depend on careful target selection and validation of sustained gene silencing in vivo, as T cells' rapid turnover presents unique challenges for siRNA therapeutics.
- Novel programs will combine validated cell-targeted LNP (ctLNP) delivery with siRNA to selectively modulate T cells in vivo
- Programs to focus on silencing hard-to-drug targets of high therapeutic value in T cell-driven autoimmune diseases
- Company reorganization supports evolution of ctLNP-enabled strategy and buildout of clinical capabilities ahead of first IND expected in 2H 2026
CAMBRIDGE, Mass., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO) a biotechnology company working to change what is possible for people living with T cell-driven autoimmune diseases, announced it is leveraging its validated T cell-directed lipid nanoparticle (ctLNP) to develop siRNA therapeutics to silence disease-driving targets in T cells.
“We are excited to move Generation Bio toward the clinic by deploying our ctLNP to deliver siRNA to T cells. By precisely modulating T cell activity in vivo we believe we can address high-value, currently undruggable targets involved in the inflammation and tissue damage associated with T cell-driven autoimmune diseases,” said Geoff McDonough, M.D., chief executive officer of Generation Bio. “Our aim is to silence therapeutic T cell targets without impacting other immune cell types, unlocking a powerful new application for siRNA in the field. We plan to submit our first IND in the second half of 2026 and to enter the clinic within our cash runway, which is into the second half of 2027.”
siRNA delivery to T cells has historically been limited by the inability to achieve selective cell targeting combined with efficient access to the cytoplasm where siRNA operates, challenges that Generation Bio has designed its ctLNP to overcome. The company recently presented non-human primate data demonstrating that its ctLNPs work by targeting T cells through a target receptor of interest with a strong selectivity for CD8+ and CD4+ effector T cells and NK cells, all of which are involved in auto-reactive tissue damage in a number of autoimmune diseases.
siRNA delivered with ctLNP aims to modulate T cell function without impacting the function of the broader immune system. ctLNP-siRNA could reach targets that change how auto-reactive T cells activate, differentiate, migrate, and damage tissues. Generation Bio will provide further details about its lead ctLNP-siRNA programs in upcoming quarters and expects to submit its first IND in the second half of 2026. The company is reorganizing to support the clinical development of T cell-directed medicines.
In addition, the company is announcing changes to its executive leadership team. Phillip Samayoa, Ph.D., chief strategy officer, will succeed Matthew Stanton, Ph.D. as chief scientific officer. Dr. Stanton will remain with the company through mid-2025 and transition to its Scientific Advisory Board thereafter. Kevin Conway, the company’s head of finance, has been elected chief financial officer to succeed Matthew Norkunas, M.D. Generation Bio also expects to appoint a chief medical officer in 2025.
“I am grateful for the passion and expertise each person at Generation Bio has contributed to enable our transition toward the clinic,” said Dr. McDonough. “Matt Stanton has been instrumental in the invention of our technologies, and I look forward to our continued work together. Likewise, Matt Norkunas has built a terrific team here, which Kevin will continue to build upon. With a strong foundation in place, we are well-positioned to execute on our mission to bring highly differentiated T cell-directed therapies to patients.”
Forward-Looking Statements
Any statements in this press release about future expectations, plans and prospects for the company, including statements about the company’s strategic plans or objectives, strategic reorganization, cash resources, technology platforms, research and clinical development plans and timelines, and preclinical data, and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the potential impact of the strategic reorganization on the company’s operations and development timeline; uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the company’s product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; uncertainties regarding the company’s novel platforms and related technologies; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; challenges in the manufacture of genetic medicine products; whether the company’s cash resources are sufficient to fund the company’s operating expenses and capital expenditure requirements for the period anticipated; as well as the other risks and uncertainties set forth in the “Risk Factors” section of the company’s most recent annual report on Form 10-K and quarterly report on Form 10-Q, which are on file with the Securities and Exchange Commission, and in subsequent filings the company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof. The company anticipates that subsequent events and developments will cause the company’s views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the company’s views as of any date subsequent to the date on which they were made.
About Generation Bio
Generation Bio is a biotechnology company changing what is possible for people living with T cell-driven autoimmune diseases. The company is developing a new modality of therapeutics leveraging its T cell selective cell-targeted lipid nanoparticle (ctLNP) to deliver siRNA in vivo, enabling modulation of T cell activity that causes inflammation and auto-reactive tissue destruction. By selectively modulating T cells that drive disease pathology, ctLNP-siRNA therapeutics could potently block target function with sequence-level specificity while sparing the broader immune system. This potent new modality is designed to reach targets that are poorly drugged by other approaches, opening a broad indication space of T cell driven autoimmune diseases.
For more information, please visit www.generationbio.com.
Investors and Media Contact
Maren Killackey
Generation Bio
mkillackey@generationbio.com
857-371-4638
FAQ
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