News and Trends 28 Feb 2023 European Commission approves first gene therapy for hemophilia B The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […] February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
Opinion 28 Feb 2023 Life changing treatments for rare disease – a beacon of hope By Sander Slootweg, managing partner, Forbion More than 300 million people around the globe are affected by a rare disease. This number does not even take into account the impact on these individuals’ support units that includes family, friends and caregivers. A rare disease is defined as a disease that affects no more than 1 […] February 28, 2023 - 6 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
Sponsored by WuXi Advanced Therapies 17 Jan 2023 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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email