Gene therapy Archives

News and Trends 23 Nov 2022

FDA approves CSL gene therapy for hemophilia B

CSL has revealed that the U.S. Food and Drug Administration (FDA) has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or […]

November 23, 2022 - 5 minutesmins - By Jim Cornall

RDEB recessive dystrophic epidermolysis bullosa skin blisters

News and Trends 21 Nov 2022

New Replay HSV gene therapy company Telaria to focus on skin diseases

Replay has announced the launch of Telaria, a herpes simplex virus (HSV) gene therapy company targeting rare skin diseases. It is the second of Replay’s product companies to leverage its high payload capacity HSV delivery vector, synHSV. Telaria’s co-founders are HSV scientists, entrepreneurs, and specialists in genetic skin diseases. In addition to Joe Glorioso, Telaria’s […]

November 21, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 16 Nov 2022

Growth in UK cell and gene therapy sector continues

The Cell and Gene Therapy Catapult (CGT Catapult) has published its ninth annual U.K. cell and gene therapy Good Manufacturing Practice (GMP) manufacturing report, showing continued growth in the advanced therapy medicinal products (ATMP) UK manufacturing sector. This growth is across industry and academia, showing how the sector is responding to the operationalization and skills […]

November 16, 2022 - 3 minutesmins - By Jim Cornall

More News! 7 Nov 2022

Sensorion granted rare pediatric disease designation by FDA for lead therapy gene

Sensorion has announced that the US Food and Drug Administration (FDA) has granted rare pediatric disease designation to its lead therapy gene candidate, OTOF-GT. The gene therapy is intended for the treatment of otoferlin gene mediated hearing loss. Sensorion’s OTOF-GT gene therapy development program aims to restore hearing in people living with otoferlin deficiency, one […]

November 7, 2022 - 2 minutesmins - By Liza Laws

In Depth 3 Nov 2022

What’s on the horizon for gene editing-based therapies?

Gene editing has come far in the past decade. However, the field is still finding its feet regarding challenges such as drug delivery, regulation and competition with first-generation gene therapies. Gene editing is a sizzling hot topic in the biotech industry, powered by advances in genetic tools such as CRISPR, TALENs and zinc-finger nucleases. Gene […]

November 3, 2022 - 8 minutesmins - By Jonathan Smith

VectorBuilder Research and Manufacturing Campus

News and Trends 2 Nov 2022

VectorBuilder to build gene delivery campus after $57M funding

Gene delivery technology company VectorBuilder Inc. has announced the completion of $57 million in funding. The funding, co-led by Legend Capital, will support the construction of VectorBuilder’s new Gene Delivery Research and Manufacturing Campus, and further boost the company’s R&D capabilities and global business presence. VectorBuilder was established in 2014 to commercialize its novel e-commerce […]

November 2, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 31 Oct 2022

Replay Bio launches HSV gene therapy company to target retinal eye disease

Replay Bio has announced the launch of Eudora, an HSV (herpes simplex virus) gene therapy company targeting genetic retinal diseases. It is the first of Replay’s product companies to leverage its high payload capacity HSV delivery vector, synHSV. Eudora’s co-founders Joe Glorioso, Mark Blumenkranz, David Schaffer, and Vinit Mahajan, are entrepreneurs who have worked in […]

October 31, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 26 Oct 2022

ABL and RD-Biotech to partner on cell and gene therapy manufacturing

French companies ABL and RD-Biotech have signed a strategic partnership to cover cell and gene therapy (C&GT) GMP manufacturing. ABL, is a contract development and manufacturing organization (CDMO) that specializes in the development and manufacturing of solutions for biopharma, including viruses for gene therapies, oncolytic viruses and vaccine candidates. RD-Biotech is a CRO (contract research […]

October 26, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 24 Oct 2022

Syncona to acquire Applied Genetic Technologies Corporation

Healthcare company Syncona Limited says its newly established portfolio company has agreed to acquire, by a tender offer, all outstanding shares of Applied Genetic Technologies Corporation (AGTC). AGTC is a clinical-stage biotechnology company focused on the development and commercialization of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an […]

October 24, 2022 - 2 minutesmins - By Jim Cornall

In Depth 20 Oct 2022

Opportunities and challenges for cell and gene therapy investments

Companies in the cell and gene therapy space are struggling to raise investments amid adverse market conditions. However, this could be an opportunity for the strongest companies and investors to shine. After several years of record-breaking investments, funding is hard to come by in the gene and cell therapy space. Part of this trend relates […]

October 20, 2022 - 9 minutesmins - By Jonathan Smith

News and Trends 13 Oct 2022

Xcell Biosciences and aCGT Vector pushing ahead with cell and gene therapies

Xcell Biosciences Inc. (Xcellbio), an instrumentation company focused on cell and gene therapy applications, and aCGT Vector, a point-of-care cell and gene therapy-as-a-service (TaaS) company, are collaborating to improve manufacturing and analytic procedures to develop personalized cell and gene therapies for cancer patients. aCGT Vector will provide its point-of-care, GMP-licensed manufacturing platform to validate Xcellbio’s […]

October 13, 2022 - 2 minutesmins - By Jim Cornall

Anastasiia Slynko biotech investment 744x368 1

In Depth 11 Oct 2022

The biggest private biotech investments in September 2022

The companies Acelyrin, InnovaFeed and Arsenal Biosciences bagged the biggest biotech investments in September 2022, with insect protein and cell therapies attracting big funding rounds. Autumn is in full swing in the Northern Hemisphere. After a breather in August 2022, September saw the return of big fundraising activity for biotech companies. We’ve gathered the biggest […]

October 11, 2022 - 3 minutesmins - By Jonathan Smith

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