Gene therapy Archives

More News! 30 Nov 2022

FDA grants Sensorion Orphan Drug Designation for hearing loss drug

Sensorion a clinical-stage biotechnology company has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to OTOF-GT, its lead gene therapy program for hearing loss. The drug is intended for the treatment of otoferlin gene mediated hearing loss. Sensorion is on track to file a clinical trial application (CTA) […]

November 30, 2022 - 3 minutesmins - By Liza Laws

News and Trends 29 Nov 2022

BioPharma Dynamics expands to provide more solutions for cell and gene therapy industry

BioPharma Dynamics have expanded their portfolio for the cell and gene therapy market to offer a range of chemically defined T-Cell media, DMSO-free cryopreservation solutions, and recombinant growth factors. Suitable for cell therapy applications at any stage, the portfolio of cell media products include immune cell media, stem cell media, and cell cryopreservation solutions. Supporting applications which […]

November 29, 2022 - 2 minutesmins - By Liza Laws

Miquel Vila Perelló gene therapy SpliceBio

Interview 28 Nov 2022

Breaking barriers to gene therapies for childhood rare diseases

The majority of known rare genetic diseases affect children, and gene therapies could help them live normal lives. Miquel Vila-Perelló, CEO and co-founder of the Spanish biotech SpliceBio, outlines how its technology could widen the range of genetic diseases treatable with gene therapies. Last week, World Children’s Day was celebrated as a means to improve […]

November 28, 2022 - 5 minutesmins - By Jonathan Smith

News and Trends 24 Nov 2022

Gene therapy approval “game changer” in hemophilia B treatment

The approval of CSL’s gene therapy Hemgenix has ushered in a new era for hemophilia B treatment. With the addition of other approvals like Rocktavian and Upstaza, 2022 could be a turning point for the broader gene therapy field. Earlier this week, etranacogene dezaparvovec (Hemgenix) made history as the first gene therapy approved by the […]

November 24, 2022 - 7 minutesmins - By Jonathan Smith

News and Trends 23 Nov 2022

BIA and Exopharm sign joint research agreement to develop integrated technology

Slovenian-based Sartorius BIA Separations (BIA), a bio-chromatography company, and Australia’s Exopharm Ltd., a company working with transformative medicines using exosomes, or extracellular vesicles (EVs), have entered into a formal collaboration. A material transfer agreement has been signed by both companies and an associated collaborative program has been implemented which aims to exploit the synergy of […]

November 23, 2022 - 4 minutesmins - By Liza Laws

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

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Gene therapy

FDA grants Sensorion Orphan Drug Designation for hearing loss drug

BioPharma Dynamics expands to provide more solutions for cell and gene therapy industry

Breaking barriers to gene therapies for childhood rare diseases

Gene therapy approval “game changer” in hemophilia B treatment

BIA and Exopharm sign joint research agreement to develop integrated technology

FDA approves CSL gene therapy for hemophilia B

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

Growth in UK cell and gene therapy sector continues

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

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

VectorBuilder to build gene delivery campus after $57M funding

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

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Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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