FDA’s draft framework would let sponsors add patient-specific genome editing variants to an approved BLA using in vitro data. Here is what the guidance proposes, what evidence it asks for, and what remains unresolved.
|Articles|August 17, 2018
Astellas Acquires Gene Therapy Company Quethera
Author(s)BioPharm International Editors
The acquisition will strengthen Astellas Pharma’s position in ophthalmology.
Advertisement
On Aug. 10, 2018, Astellas Pharma announced that it has acquired Quethera, a Cambridge, United Kingdom-based gene therapy company focused on developing new treatments for ocular disorders, including glaucoma.
Under the terms of the agreement, Astellas may pay up to £85 million (US$108 million) in upfront and contingent payments to Quethera shareholders. Upon the closing of the transaction, Quethera has become a wholly owned subsidiary of Astellas.
Through this transaction, Astellas has acquired Quethera's ophthalmic gene therapy program, which uses a recombinant adeno-associated viral vector system (rAAV) to introduce therapeutic genes into target retinal cells for the treatment of glaucoma. According to Astrellas, the lead pre-clinical candidate of the program has demonstrated significantly improved survival of retinal ganglion cells in pre-clinical models.
"We believe the rAAV program has potential as a new therapeutic option for the treatment of refractory glaucoma through an intraocular pressure-independent mechanism,” said Kenji Yasukawa, PhD, president and CEO, Astellas Pharma, in a company press release. “It would address a high unmet medical need in glaucoma patients who are at risk of losing their eyesight."
“This deal enables us [at Quethera] to accelerate our evaluation of this investigational technology program to see if we can slow or prevent disease progression for these patients," said Peter Widdowson, PhD, CEO, Quethera, in the release.
Source:
Advertisement
Related to this article
In the first part of a two-part interview, Paz Vellanki of Precision for Medicine explains what sponsors should bring to early FDA meetings and how manufacturing and assay readiness can delay rare disease trials.
FDA clears the IND for Biolojic Design’s BD200, an AI-designed Trop2/Nectin-4 multibody ADC; Blackstone Life Sciences agrees to provide Pfizer up to $1.5 billion to develop its monthly GLP-1 receptor agonist berobenatide; and enGene Therapeutics plans a year-end BLA for detalimogene voraplasmid in BCG-unresponsive non-muscle invasive bladder cancer.
FDA has cleared Biolojic Design’s IND for BD200, a bispecific antibody-drug conjugate targeting Trop2 and Nectin-4 whose arms can each bind either target. A global Phase 1 study in solid tumors is expected to begin in the coming weeks.
Blackstone Life Sciences will fund up to $1.5 billion of development costs through 2028 for Pfizer’s monthly GLP-1 receptor agonist berobenatide, in exchange for milestone payments tied to approval and sales. Pfizer keeps full control of the program.
Beam Therapeutics’ base-edited cell therapy risto-cel meets its primary endpoint in the BEACON sickle cell disease trial, Amgen’s CD40L antagonist dazodalibep reduces systemic disease activity in the phase 3 OASIZ-301 Sjögren’s disease trial, and ImvivaBio’s allogeneic CD19/BCMA CAR-T CTA313 sustains lupus remission through 12 months.
ImvivaBio’s off-the-shelf CD19/BCMA CAR-T cell therapy CTA313 kept 88% of evaluable patients with refractory lupus in DORIS remission at Month 12, according to longer-term phase 1 data presented at ACR Convergence 2026.
Full phase 3 OASIZ-301 results show both dosing regimens of Amgen’s CD40L antagonist fusion protein dazodalibep reduced systemic disease activity in Sjögren’s disease at Week 48. The every-four-weeks dose also improved patient-reported dryness.
Advertisement
Advertisement
Trending on BioPharm International
1
ImvivaBio’s Dual-Target Allogeneic CAR-T Sustains Lupus Remission Through 12 Months
2
Updates on Simtra BioPharma Solutions’ Bloomington Capacity Expansions
3
Blackstone to Provide Pfizer Up to $1.5 Billion for Monthly GLP-1 Berobenatide Development
4
Amgen’s Dazodalibep Reduces Systemic Disease Activity in Phase 3 Sjögren’s Trial
5