After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy, valoctocogene roxaparvovec, following a rejection in August 2020. It's armed with data it hopes will assuage concerns about long-term safety and benefits.
Recognizing that academic sponsors and nonprofits are major contributors to the development of advanced therapy medical products (ATMPs) and diagnostic and delivery devices, the EMA is launching a pilot program to help them navigate the challenging regulatory requirements in the space.
Bluebird Bio Inc.’s elivaldogene autotemcel (eli-cel) gained U.S. approval late Sept. 16 for use in early active cerebral adrenoleukodystrophy (CALD), making it the firm’s second gene therapy to clear the FDA in as many months. Branded Skysona, eli-cel is expected to be available commercially by the end of 2022 and its launch will require only “incremental” company resources on top of those required for the ongoing launch of beta-thalassemia gene therapy Zynteglo (betibeglogene autotemcel), Bluebird said.
Ocular gene therapy firm Sparingvision SA raised €75 million (US$75 million) in a series B round to fund its transition to clinical development. The company is about to move its lead program, the mutation-agnostic gene therapy SPVN-06, into a phase I trial in retinitis pigmentosa (RP). “We are in the middle of the regulatory submission process,” CEO Stéphane Boissel told BioWorld.
The University of Sydney is investing AU$478 million (US$326.5 million) to build a leading biomedical precinct to fast-track research and shorten the time between discovery and development of transformative therapies.
Epigenic Therapeutics Co. Ltd., a company developing epigenomic-focused gene therapies in multiple indications, has raised $20 million across angel and pre-series A rounds to support preclinical studies, early stage research, and growth for its team.
Bluebird Bio Inc. isn’t giving out much of the information on the margins for the cost of its newly approved cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. The numbers will, the company said, come into better focus when another Bluebird drug is approved and launched.
The U.S. FDA has approved the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia requiring frequent red blood cell transfusions. The $2.8 million wholesale acquisition cost for the one-time I.V. infusion will make it one of the most expensive drugs in the U.S.
Vector Biopharma AG has secured a $30 million series A funding commitment from founding investor Versant Ventures to take forward a new gene delivery platform developed by Andreas Plückthun, of the University of Zurich, in Switzerland.
Increased payload capacity for gene therapies, off-the-shelf genome-engineered allogeneic cell therapies, reduced cost of goods and faster bioprocesses, are promised by “big DNA” specialist Replay Holdings LLC. The newco arrived with a $55 million seed round and having assembled a portfolio of technologies for writing and delivering large pieces of DNA.