MeiraGTx secures up to $400 million to accelerate commercialisation
Gene therapy developer MeiraGTx has secured up to $400 million through healthcare investment firm Oberland Capital, providing substantial capital to advance its ophthalmology pipeline and support commercial preparations. The agreement includes an upfront investment with additional milestone-based funding, giving the company flexibility to continue development while avoiding the significant shareholder dilution associated with traditional equity financing.
The funding will primarily support three of MeiraGTx’s eye disease candidates and shows a vote of confidence in ophthalmic gene therapy at a time when several high-profile programmes in the space have been cut. Royalty financing structures like this are becoming an increasingly important funding route for CGT companies with de-risked, late-stage assets but limited access to traditional capital markets. For the sector, this is a signal that investors are willing to support programmes with compelling clinical data and clear commercial potential.
Read more via BiopharmaDive
J&J discontinues inherited retinal disease gene therapy programme
Johnson & Johnson has confirmed it is discontinuing development of its AAV2-based gene therapy for geographic atrophy, following disappointing Phase IIb results. The therapy failed to demonstrate sufficient efficacy compared with the control arm, leading the company to stop further development of the programme. A J&J spokesperson confirmed the programme's discontinuation following an assessment of the topline data, noting that learnings will feed into the company's early-stage ophthalmology pipeline. The news comes alongside J&J's confirmation that it has also removed bota-vec (sold to MeiraGTx earlier this year) and two CAR-T candidates from its pipeline.
Geographic atrophy remains a serious unmet need with no cure, and this is now the second major setback for AAV-based complement-pathway approaches in the indication. Pipeline attrition remains an inevitable part of developing advanced therapies however, these decisions also reflect increasing portfolio discipline across pharmaceutical companies.
Read more via Fierce Biotech
ARPA-H awards up to $38.9 million to accelerate personalised gene editing
Children's Hospital of Philadelphia (CHOP) has been awarded up to $38.9 million over five years from ARPA-H's THRIVE programme, the largest single award in a $160 million initiative aimed at turning bespoke gene-editing therapies into a scalable treatment platform.
The programme aims to create a scalable framework capable of designing, manufacturing and delivering bespoke CRISPR-based medicines tailored to individual patients with life-threatening threatening conditions. Rather than treating one disease at a time, researchers hope to establish a repeatable development platform that could dramatically reduce the time required to produce personalised therapies.
This initiative builds on growing momentum around “n-of-1” medicines, where therapies are developed specifically for individual patients with unique genetic mutations. Significant public investments from organisations such as ARPA-H demonstrates growing confidence that manufacturing and commercial barriers can be overcome through platform-based approaches. If successful, the programme could help establish a blueprint for delivering personalised genetic medicines at scale, opening treatment options for thousands of patients with diseases that would otherwise remain commercially unviable.
Read more via Morning Star
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