Author: Juliet Preston

Capitalizing on a gene therapy boom, Avrobio locks in $60M

Cambridge, Massachusetts-based Avrobio has closed an oversubscribed $60 million Series B financing round to further its ex vivo lentiviral platform for the treatment of lysosomal storage disorders. It’s one more sign that after decades of ups and downs, gene therapies have finally arrived. Announced Thursday, the raise was co-led by Cormorant Asset Management and Surveyor Capital, with support from Aisling, Brace Pharma Capital, Eventide, Morningside and Leerink Partners. Early backers Atlas Venture, SV Health Investors, and Clarus Ventures also chipped in. It follows a successful Phase 1 trial for Avrobio’s lead program in Fabry disease in 2017. While it...

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Gene editing remains early stage, but Big Pharma has brought notice

The field of gene modifying is moving fast. In late October, two new documents described technologies that allow for the precise editing of individual DNA bases  ? an A to a T, the C to a G? and, separately, a mechanism regarding genetically editing RNA . Both have been heralded as main advances. But let’ s back up a bit from the cutting edge tools to the actual application: How is gene editing progressing, in terms of biotech pipelines, investment, and therapeutic approvals? In its recent report, titled Gene Editing: The Next Breakthrough within Regenerative Medicine , Informa Pharma...

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Reinventing tissue regeneration, one layer at a time

There are some fancy tools out there for repairing skin, from 3D bioprinting, scaffolds and matrices to spray guns that rain stem cells directly onto a wound. Doctors in Brazil are even experimenting with sterilized Tilapia fish skin as a novel dressing for burns. Creativity is nice, but that alone won’t save patients battling through the most critical hours of their lives. Denver Lough, an M.D./Ph.D., saw this first hand while working at the Johns Hopkins Plastic and Reconstructive Surgery Program. Nothing was truly getting the job done. “Name one regenerative medicine product or company that’s out there that actually truly regenerates anything,” Lough challenged in a recent phone interview. “Not, ‘we grow keratinocytes,’ or ‘we can turn a cell into an osteogenic lineage’ but really; does this grow full thickness tissue? There’s really not one out there and there’s certainly not one that’s being used clinically right now for skin regeneration.” Now CEO and CSO of PolarityTE, Lough believes the fundamental approach to tissue regeneration is taught wrong. And that’s why, after all these years, skin healing remains imperfect. Biotechs working in this field have zoomed in on individual cells, he said, working to culture and manipulate stem cells or to find the right recipe for growth factors that can guide differentiation. That’s not how biology works, Lough contests. “[Biology] works through cells interacting with each other, having...

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It’s not a competition but… Kite’s CAR-T therapy just claimed a first in Europe

As Novartis awaits a final FDA ruling on its CAR-T therapy, Santa Monica, California-based Kite Pharma has jumped the Atlantic to file the first Marketing Authorization Application (MAA) with the European Medicines Agency (EMA). Announced Monday, the MAA centers around axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor (CAR) T-cell therapy that targets CD19, a protein expressed on the surface of the B-cells that drive lymphomas and leukemias. Axi-cel is being pitched as a potential treatment for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) who are ineligible for autologous stem cell transplants. It also has a biologics submission filed with FDA, though it is several months behind Novartis. While it seems inevitable that one of the CAR-T therapies will soon reach the market, many question marks remain around the post-marking logistics — domestically and internationally. Kite unveiled an early outline for manufacturing and distribution of the therapy at the J.P. Morgan Healthcare Conference in January. The company has reportedly constructed a manufacturing facility in El Segundo, California, in close proximity to the Los Angeles International Airport (LAX). T-cells harvested from patients throughout the U.S. would be flown there for engineering, before being sent back to the patient’s local clinic for reinfusion. According to E.P. Vantage’s Jacob Plieth, the El Segundo facility would also service Europe. $KITE just confirmed to me that KTE-C19 manufacturing under...

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Why the immune system is our best bet to battle cancer

It’s a big week for oncology and immunotherapy, as an FDA panel convenes on Wednesday to interrogate the first CAR-T therapy vying for approval. Developed by Novartis, the five-step approach seeks to harness and weaponize the patient’s own immune system to fight off late-stage blood cancers. Kite Pharma has its own version awaiting FDA review and several others are moving through clinical trials. With their almost science fiction-like complexity and remarkable effects, Chimeric Antigen Receptor-engineered T-cells (CAR-Ts) have understandably captured the field’s attention. But underneath the hype and excitement, all the different programs rely on a much more basic and familiar aspect of cell biology: the protein/receptor target. Whether it’s a cancer vaccine, an antibody-drug conjugate (ADC), or an entirely new platform; there has to be a way for the therapy and/or the immune system to differentiate between cancerous and healthy cells. It’s a common denominator throughout all immuno-oncology programs and as a result, it can be a window into the evolution and expansion of the field. All or nothingAccording to a recent report by the trade group PhRMA, there are 240 cancer immunotherapies in the pipeline. For each candidate, R&D teams have to ask the same fundamental question: How can we drive or enable an immune response? The lead programs of both Novartis and Kite are actively programming T-cells to seek out CD19, a protein found on the surface of...

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