Gene therapy Archives

News and Trends 17 Feb 2023

Report: China leads the way with phase 1 studies

Novotech, an Asia Pacific biotech CRO, has published a new global report on phase 1 trial activity including current metrics on fast growth regions and therapeutic areas. The data analysis found that Asia Pacific has been the fastest-growing region for phase 1 clinical trials in the last 10 years, reaching a share of 58% in […]

February 17, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to RP-A501. RP-A501 is Rocket Pharmaceuticals’ investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 9 Feb 2023

Cell and Gene Therapy Catapult boost for life science campus

The Cell and Gene Therapy Catapult (CGT Catapult), an independent U.K. innovation and technology organization specializing in the advancement of cell and gene therapies, has announced its involvement in the development of a major new campus for life science businesses in Stevenage. The new campus was announced by UBS Asset Management’s Real Estate & Private […]

February 9, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 31 Jan 2023

Sensorion and EVEON collaborating on injection system to deliver gene therapy inner ear treatments

Sensorion and EVEON are collaborating on the design and development of an injection system for the delivery of Sensorion’s gene therapy products to the inner ear. Therapies for treating genetic hearing loss are often challenging due to the need for delivery into the inner ear, a very small and delicate organ embedded in the temporal […]

January 31, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 24 Jan 2023

Funding for university to develop new motor neuron disease therapies

Medical research charity LifeArc, the Motor Neurone Disease Association (MND Association) and the My Name’5 Doddie Foundation have jointly awarded £1 million ($1.2 million) to researchers at University College London (UCL) to progress two pioneering new therapies that could help transform the lives of those living with motor neuron disease (MND). The research grants are […]

January 24, 2023 - 4 minutesmins - By Jim Cornall

In Depth 17 Jan 2023

Cell and gene therapies gain momentum in Basel

Companies specializing in the research and development of innovative cell and gene therapies continue to spring up all around the globe, and a growing number are choosing the Swiss city of Basel as their base. These companies are taking advantage of the talent pool, academic institutions and investors that the area has to offer, culturing […]

January 17, 2023 - 5 minutesmins - By External Contributor

News and Trends 17 Jan 2023

TTP spinout Cellular Origins to focus on cell and gene therapies

TTP plc, a technology and product development company, has announced the launch of Cellular Origins. Cellular Origins is a TTP spin-out, created to enable scalable, cost-effective and efficient manufacture of cell and gene therapies. Cellular Origins’ proprietary technology addresses the current barriers associated with the manufacture of advanced therapies that are in late-stage development, enabling […]

January 17, 2023 - 3 minutesmins - By Jim Cornall

Navigating cell and gene therapy manufacturing with a long-term partnership

Cell and gene therapies have proven to be promising treatments for cancers and genetic diseases, but developers face key challenges before these treatments reach patients’ bedsides. A long-term partnership with a contract development and manufacturing organization (CDMO) can ease the path life science companies travel from manufacturing these life-changing treatments and bringing them to the […]

January 17, 2023 - 7 minutesmins - By Alexander Burik

News and Trends 12 Jan 2023

Team wins $149M contract for National Institute of Neurological Disorders and Stroke

A team consisting of Battelle, AmplifyBio and Andelyn Biosciences has won a seat on an eight-year, $149 million indefinite delivery, indefinite quantity (IDIQ) contract vehicle for the National Institute of Neurological Disorders and Stroke in the U.S. As one of the prime contractors on the multi-award IDIQ, the team will provide manufacturing and nonclinical support […]

January 12, 2023 - 3 minutesmins - By Jim Cornall

Woman looking through a window and seeing biotechnology such as pills and vaccines. It indicates the biotech trends coming in the future

Best in Biotech 12 Jan 2023

The biggest biotech trends that will shape 2023

2023 has begun at full throttle. We have collected seven major biotech trends that industry experts are keeping an eye on over the next 12 months. Just like that, another year has begun. 2023 has already launched with announcements of big pharma acquisitions and the approval of Eisai and Biogen’s Alzheimer’s drug lecanemab by the […]

January 12, 2023 - 9 minutesmins - By Jonathan Smith

News and Trends 11 Jan 2023

Clinical trends for 2023

By Richard Staines, account director, Optimum Strategic Communications Few would deny that the decade has had a turbulent start, but after the tragedies of the COVID-19 pandemic and the Ukraine war, there’s hope that 2023 will see a new wave of clinical research. Exciting new technologies such as gene editing, gene therapy and a next […]

January 11, 2023 - 8 minutesmins - By External Contributor

News and Trends 9 Jan 2023

Selecta and Astellas sign LOPD treatment agreement

Selecta Biosciences, Inc. and Astellas Pharma Inc. have announced an exclusive licensing and development agreement for IdeXork (Xork). Xork is being studied as a potential next generation immunoglobulin G (IgG) protease that will be developed by Astellas for use with AT845, an investigational, adeno-associated virus (AAV)-based treatment for late-onset Pompe disease (LOPD) in adults. Pompe […]

January 9, 2023 - 3 minutesmins - By Jim Cornall

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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

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Gene therapy

Report: China leads the way with phase 1 studies

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Cell and Gene Therapy Catapult boost for life science campus

Sensorion and EVEON collaborating on injection system to deliver gene therapy inner ear treatments

Funding for university to develop new motor neuron disease therapies

Cell and gene therapies gain momentum in Basel

TTP spinout Cellular Origins to focus on cell and gene therapies

Navigating cell and gene therapy manufacturing with a long-term partnership

Team wins $149M contract for National Institute of Neurological Disorders and Stroke

The biggest biotech trends that will shape 2023

Clinical trends for 2023

Selecta and Astellas sign LOPD treatment agreement

Subscribe to our newsletter to get the latest biotech news!

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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