Roche discontinues Poseida’s allogeneic CAR-T candidate amid pipeline review
Roche has discontinued development of RG6540, an allogeneic CAR-T therapy acquired through its $1.5 billion takeover of Poseida Therapeutics, as part of a wider review of its early-stage pipeline. RG6540 is an off-the-shelf CAR-T candidate designed to target both CD19 and CD20, with Phase 1 clinical programs previously evaluating its potential in B-cell malignancies and autoimmune conditions.
Despite the discontinuation, Roche CEO Thomas Schinecker has maintained that the company's position on the Poseida acquisition remains unchanged, confirming that Roche continues to advance alternative CD19xCD20 CAR-T candidates originating from the acquired platform.
The decision comes amid a broader reassessment of allogeneic CAR-T across the industry, shortly after news of Caribou cutting its off the shelf pipeline. As developers continue to evaluate the clinical and commercial potential of allogeneic therapies, it raises a broader question question: With further allogeneic programs falling out of development, is the industry's vision of an off-the-shelf CAR-T future becoming increasingly difficult to realise?
Read more via BioSpace
Estuary Biotherapeutics launches with $103M to advance gene therapies for kidney disease
Estuary Biotherapeutics has emerged from stealth with $103 million in committed financing to advance a pipeline of targeted gene therapies for autosomal dominant polycystic kidney disease (ADPKD), one of the most common inherited kidney disorders.
The financing comprises a $93 million Series A led by RA Capital Management, alongside an earlier $10 million seed round co-led by 4BIO Capital and GV. Additional investors include Deep Track Capital, Biogen New Ventures and UPMC Enterprises.
Founded on research originating from Yale School of Medicine, Estuary is developing locally delivered adeno-associate virus (AAV) gene therapies designed to address the underlying genetic causes of ADPKD, rather than solely managing disease progression.
Its two lead candidates, EST-201 and EST-101, target mutations in the PKD2 and PKD1 genes, respectively. Both programs use AAV-based delivery to restore functional polycystin protein expression in kidney cells, with the aim of providing durable therapeutic benefit.
Estuary expects to advance EST-201 into clinical development in 2027, followed by EST-101 in 2028. Proceeds from the financing will support both programs through clinical proof of concept, alongside further development of the company’s kidney-targeting delivery platform.
The launch represents a significant investment in extending gene therapy into kidney disease, where effective delivery to the target organ remains a major development challenge. Estuary's approach seeks to overcome this barrier through localised administration, potentially opening a new treatment pathway for patients with genetically defined forms of ADPKD
Read more via The Pharma Letter
Opus Genetics receives FDA clearance for childhood blindness in gene therapy
Opus Genetics has received clearance from the US FDA for its Investigation New Drug (IND) application for OPGx-RDH12, enabling the company to advance its investigational gene therapy into clinical development.
The candidate is being developed for RDH12-associated Leber congenital amaurosis (RDH12-LCA), a rare inherited retinal disorder characterized by progressive vision loss beginning in early childhood. OPGx-RDH12 is an AAV-based gene therapy designed to deliver a functional copy of the RDH12 gene directly to photoreceptor cells in the retina. Preclinical studies in cell and mouse models have demonstrated restoration of RDH12 activity and functional improvements, providing the basis for its progression into clinical evaluation.
Opus plans to initiate a Phase 1/2 clinical trial in the fourth quarter of 2026, marking the third gene therapy program from its pipeline to enter clinical development. The program is being advanced in collaboration with the Global RDH12 Alliance, comprising US-based RDH12 Fund for Sight and UK-based Eyes on the Future. Established in July 2025, the partnership includes a commitment of up to $1.6 million towards development costs, alongside collaboration on the clinical and regulatory strategy.
The IND clearance marks an important milestone for the program and highlights the role of patient advocacy organizations in supporting the development of gene therapies for rare diseases, particularly where small patient populations and limited commercial incentives can present additional development challenges.
Read more via Globe Newswire
Marker Therapeutics secures $2.6M FDA grant to advance pancreatic cancer cell therapy
Marker Therapeutics has been awarded a $2.6 million grant from the FDA's Orphan Products Clinical Trials program to support the clinical development of MT-601, its investigational multi-antigen recognizing T-cell (MAR-T) therapy, in pancreatic cancer. The funding will support a Phase 1 study evaluating MT-601 in patients with locally advanced, unresectable pancreatic cancer, with clinical initiation anticipated in the first quarter of 2027.
Unlike conventional CAR-T therapies, Marker's MAR-T platform uses non-genetically modified T cells capable of recognizing multiple tumour-associated antigens. MT-601 is designed to target six different antigens, with the aim of addressing tumour heterogeneity and reducing the likelihood of antigen escape, two significant challenges in developing effective cell therapies for solid tumours.
The planned study builds on earlier Phase 1/2 research conducted at Baylor College of Medicine, which reported a disease control rate of 84.6% in patients receiving multi-antigen targeted T cells alongside frontline chemotherapy. The study also reported a median duration of response of 7.5 months and median overall survival of 14.1 months.
The award was one of six projects selected from 66 applications submitted to the FDA's Orphan Products Clinical Trials grants program in fiscal year 2026. It provides Marker with additional non-dilutive funding to advance MT-601, while supporting the continued evaluation of multi-antigen T-cell therapies in solid tumors, an area where conventional cell therapy approaches have faced considerable clinical challenges.
Read more via Marker Therapeutics
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