Just hours after J&J's oncology team bragged about scoring a breakthrough therapy designation for their BCMA CAR-T drug, they pulled the wraps off of the multiple myeloma data for ...
Decrying 'arbitrary and capricious' action, RegenxBio sues FDA over clinical holds on gene therapy
When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.
As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.
RegenxBio runs into partial hold for anti-VEGF gene therapy as FDA investigates delivery device
Issues with a delivery device are slowing down RegenxBio’s clinical plans for one of its lead gene therapies.
In its Q3 update, RegenxBio informed investors of a partial clinical hold the FDA has slapped on RGX-314 while regulators review certain third-party commercially-available devices the biotech uses to deliver its treatment. As a result, RegenxBio won’t be able to start its planned Phase IIb trial for wet age-related macular degeneration or file an IND for a Phase II diabetic retinopathy study until early 2020.
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Thermo Fisher hooks gut onto its gene therapy pipeline, inks new collaboration pact
Seven months ago, Thermo Fisher spent $1.7 billion to add a gene therapy company to its sprawling biotech business, purchasing Brammer Bio. Now they’ve signed a collaboration deal with another, newly well-funded, company from the same founder to expand its gene therapy work into the microbiome.
Thermo Fisher announced a collaboration with microbiome product manufacturer Arranta Bio as the startup announced an $82 million funding round. The young CDMO will provide its live biopharmaceuticals to Thermo Fisher to use in gene therapy production.
Novartis takes a hit as FDA orders a hold on Zolgensma trials in wake of fresh safety concerns
The FDA has ordered Novartis to halt enrollment and dosing in a set of trials for Zolgensma, their gene therapy for lethal cases of spinal muscular atrophy. And the latest setback triggered a frenzy of analyst notes as they sized up who among Novartis’ rivals in the field would benefit most.
The hold was implemented after Novartis’ subsidiary AveXis — still operating under the glare of an FDA investigation into an ethics scandal — informed regulators about concerns raised by an animal study pointing to “dorsal root ganglia (DRG) mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss.”
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Sean Parker helps create a CRISPRed cell therapy 2.0 play — and he’s got a high-profile set of leaders on the team
You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. It’s another appealing Bay Area group that’s attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.
Today it’s Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenberg’s new venture investment operation based in LA.
Drazan — a J&J Innovation vet with a long record of entrepreneurial endeavors — exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasn’t long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.
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Exclusive: GSK’s Hal Barron allies with Rick Klausner’s $600M cell therapy startup, looking to break new ground blitzing solid tumors
LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.
Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.
“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”
“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”
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Ness Bermingham. Korro Bio
October 7, 2019 07:07 AM EDTUpdated October 8, 11:15 AM
Ness Bermingham looks to lead the wave of RNA editing at new Atlas upstart Korro Bio
By the time Ness Bermingham left Intellia in late 2017, the gene-editing biotech had secured its footing as one of the pioneers in the CRISPR/Cas9 space with a highly anticipated first-in-human trial in the works. Returning to his VC post at Atlas Venture, he signaled that he was looking to do it all over again with the next big startup idea.
And now he’s ready to talk about that venture, which represents the latest evolution of his longstanding interest in tinkering with the genome — RNA editing.
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Early functional data on limb girdle patients buoy expectations for Sarepta's gene therapy
Sarepta’s lousy summer has come to a close — with some encouraging fresh functional data from its limb-girdle muscular dystrophy (LGMD) gene-therapy program.
In February, the drugmaker divulged data from three patients in the first cohort of an open-label Phase I/II study testing the use of an experimental gene therapy — MYO-101 (now called SRP-9003) — in LGMD patients aged four to 15 years.
Data showed the therapy rejuvenated the production, by an average of 51%, of beta-sarcoglycan — the protein required for muscle function that is missing in this patient population. On Friday, Sarepta broke out data that showed that the induced uptick in beta-sarcoglycan did, in fact, translate to functional improvements, nine months post-infusion.
Based on the natural history of the disease, these changes were definitely not expected at these time points, Sarepta executives underscored on a conference call with analysts.
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Regeneron doubles down on partner Adicet's gamma delta T cell tech in $80M financing
Three years after singling out Adicet Bio as a top contender in the race for a new wave of off-the-shelf immune cell therapies and putting down $25 million upfront for a research pact, Regeneron is reinforcing its endorsement in the form of an equity investment.
The big biotech has jumped into an $80 million Series B for its partner, which is designed to further its work on gamma delta T cells and push a lead therapy for non-Hodgkin’s lymphoma to the clinic. Solid tumor programs will follow.
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San Francisco biotech has its heart in heart failure — and $92M in the bank
When a salamander’s heart suffers damage, the amphibian’s cells are able to divide and repair the organ. Tenaya Therapeutics wants to empower your heart with the same ability.
The South San Francisco-based company, founded by scientists at the Gladstone Institutes and University of Texas Southwestern Medical Center in October 2016, has a multi-layered approach to addressing troubles of the heart — cellular regeneration, gene therapy, and precision medicine.
