Gene therapy approval, Sangamo’s asset sale and new iPSC therapy enters the clinic – Phacilitate Weekly News Round-up

Ultragenyx secures first FDA approval with gene therapy for GSDIa

Ultragenyx has received its first FDA approval for a gene therapy, with the agency granting accelerated approval to Genglycos (pariglasgene brecaparvovec-opnr) for the treatment of glycogen storage disease type Ia (GSDIa) in adults and children aged eight and older. The approval marks an important milestone for the company and comes just over a year after the FDA rejected another Ultragenyx gene therapy candidate over manufacturing related concerns.

GSDIa is an ultra-rare genetic disorder caused by a deficiency in glucose-6-phosphatase, an enzyme required for the liver to release glucose into the bloodstream during fasting. Genglycos is a one-time AAV8 gene therapy designed to address the underlying genetic defect by delivering a functional copy of the G6PC gene directly to liver cells.

The accelerated approval means Ultragenyx will be required to generate additional post-marketing evidence. The company plans to follow 50 commercially treated patients for two years, alongside 20 patients who cannot receive the therapy because of pre-existing AAV8 antibodies. Ultragenyx will manufacture Genglycos at its gene therapy facility in Bedford, Massachusetts, while its UltraCare programme will provide support around treatment logistics and insurance coverage. The approval also comes with a Priority Review Voucher, giving Ultragenyx an additional potentially valuable regulatory asset. The company is now preparing to launch its first commercial gene therapy, while its previously rejected UX111 programme for Sanfilippo syndrome remains under FDA review.

Read more via Fierce Pharma

PTC Therapeutics and Eli Lilly acquire key Sangamo assets

Sangamo Therapeutics has completed a competitive auction for several of its core assets following its bankruptcy filing, with PTC therapeutics and Eli Lilly emerging as the major buyers.

PTC has agreed to acquire Sangamo’s Fabry disease gene therapy programme, isaralgagene civaparvovec (ST-920), for $111 million upfront and up to a further $100 million in potential milestones. The deal represents a significant increase on the $25 million upfront offer previously agreed with Astellas.

ST-920 is already at the BLA stage, with Sangamo having begun a rolling submission to the FDA. PTC expects to complete the filing in the fourth quarter, with $80 million of the potential milestone payment tied to accelerated FDA approval and a further $20 million linked to full approval. PTC also plans to pursue regulatory approvals outside the US. For PTC, the acquisition provides an opportunity to enter the Fabry disease market using an asset that is already relatively advanced. The company said the programme was particularly attractive because it could leverage existing infrastructure, capacity and expertise without requiring significant additional investment.

Eli Lilly, meanwhile, has agreed to pay $50 million for Sangamo's genomic medicine technology platforms, including its capsid delivery, zinc finger and modular integrase technologies, alongside ST-506, an investigational epigenetic regulator for prion disease. Other Sangamo assets, including programmes in haemophilia A, chronic neuropathic pain and cell therapy, remain available for sale.

Subject to bankruptcy court approval, proceeds from the transactions will be distributed to creditors as Sangamo moves towards winding down the company. The auction highlights the potential value that can remain embedded in advanced therapy platforms and clinical-stage assets even when a biotech is unable to continue funding their development independently.

Read more via pharmaphorum

Sumitomo Pharma doses first patient with iPSC-derived retinal therapy

Sumitomo Pharma America has dosed the first patient in a Phase 1/2a study of DSP-3077, an investigational allogeneic iPSC-derived retinal cell therapy for non-syndromic retinitis pigmentosa (RP). The first patient has undergone a subretinal implantation of the therapy, marking an important step for the company's regenerative medicine programme.

DSP-3077 consists of multilayered retinal sheets generated from induced pluripotent stem cells. The therapy is based on a three-dimensional retinal organoid produced using a self-organising cell culture technique and contains retinal tissue structures including photoreceptor precursors. The approach aims to replace or support retinal cells lost through the progressive degeneration associated with RP.

The open-label, single-arm study will evaluate two dose levels in adults with RP. Across three cohorts, 12 patients will receive a single subretinal injection in one eye. The primary objectives are to assess safety and tolerability, with secondary assessments focused on engraftment, potential therapeutic response and performance of the delivery device.

The first patient treatment adds to growing clinical interest in iPSC-derived regenerative medicine and demonstrates how advances in cell manufacturing and tissue engineering are beginning to translate into clinical development programmes targeting diseases where conventional gene replacement approaches may be difficult to apply.

Read more via Clinical Trials Arena