Gene therapy news - the latest biotech news from Labiotech.eu

Genetic engineering giants: is China poised to lead the way?

Willow Shah-Neville — Wed, 19 Jul 2023 15:57:04 +0000

For many people, when they hear China and genetic engineering in the same sentence, it is often synonymous with scandal, and gene-edited babies may spring to mind. And, although it is true that nearly five years ago, researcher He Jiankui infamously claimed he had created the first ever gene-edited babies, before going to prison for […]

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Gene therapy company EG 427 raises €18M

Jim Cornall — Thu, 13 Jul 2023 08:30:00 +0000

EG 427, a French biotechnology company leading the development of pinpoint DNA medicine solutions, has announced the final closing of a Series A financing. The company raised an additional €5 million ($5.6 million) in the final closing from a combination of existing investors and new family office investors, bringing the total raised in the Series […]

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NK:IO awarded £1.6M grant to develop NK cell therapy

Jim Cornall — Tue, 11 Jul 2023 09:45:14 +0000

NK:IO, a U.K. company working on natural killer (NK) cell biology applied to the development of exceptionally potent, off-the-shelf cell therapies targeting solid tumors, has been awarded £1.6 million ($2.1 million) in grant funding from Innovate UK’s New Cancer Therapeutics program. The funding will support preclinical development, including development of a manufacturing process in collaboration […]

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FDA approves BioMarin gene therapy for hemophilia A

Jim Cornall — Fri, 30 Jun 2023 08:21:23 +0000

BioMarin Pharmaceutical Inc. has announced that the United States Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The […]

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Astraveus exits stealth to advance cell and gene therapy platform

Jim Cornall — Tue, 27 Jun 2023 09:00:00 +0000

Astraveus SAS has emerged from stealth with the completion of an oversubscribed €16.5 million ($18.1 million) Series Seed financing. The company creates modular, microfluidic cell foundries that transform cell and gene therapy (CGT) manufacturing. Astraveus has developed the Lakhesys platform, an end-to-end cell foundry that uses deep process optimization and single-use, microfluidic bioprocessors to deliver […]

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Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy

Jim Cornall — Fri, 23 Jun 2023 08:45:00 +0000

Sarepta Therapeutics, Inc. has announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an Adeno-associated virus (AAV)-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval […]

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Gene therapy: a promising solution for sickle cell disease

Willow Shah-Neville — Mon, 19 Jun 2023 10:00:00 +0000

Sickle cell disease is debilitating, and many people who suffer with it find that their lives are defined by pain. This means a curative treatment would be completely life–altering, and, as we observe World Sickle Cell Day on June 19, we explore how gene therapy could be the most promising solution for curing sickle cell […]

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Beyond Biotech podcast 50

Jim Cornall — Fri, 16 Jun 2023 09:45:31 +0000

The International Society for Cell & Gene Therapy (ISCT), the global society of more than 3,000 members dedicated to the translation of cell and gene therapies (CGT) into safe and effective treatments to improve patient lives, recently hosted the largest translation-focused event connecting academic, regulatory and industry experts from around the world to collaborate and […]

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Kate Therapeutics launches with $51M to develop genetic medicines for muscle and heart diseases

Jim Cornall — Thu, 08 Jun 2023 09:45:00 +0000

Gene therapy company Kate Therapeutics Inc. (KateTx), has emerged from stealth mode with a $51 million Series A financing. In addition, the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM). There are a large number of genetically defined and complex muscle […]

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AAVantgarde closes €61M financing to move gene delivery program to the clinic

Jim Cornall — Wed, 07 Jun 2023 08:47:46 +0000

AAVantgarde Bio, a clinical-stage, Italian-based international biotechnology company with two proprietary adeno-associated viral (AAV) vector platforms for large gene delivery, has closed a €61 million ($65.1 million) Series A financing. The amount represents the largest series A round ever in Italian biotech. AAVantgarde has two proprietary, AAV-based large gene delivery platforms: one leveraging DNA recombination, […]

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ElevateBio raises $401M for cell and gene therapies

Jim Cornall — Thu, 01 Jun 2023 09:30:00 +0000

ElevateBio, LLC (ElevateBio), a technology-driven company focused on cell and gene therapies, has closed its $401 million Series D financing. “We have made significant strides in scaling our technologies and end-to-end capabilities in our pursuit to become the world’s most indispensable cell and gene therapy technology company. We are thrilled to welcome Khalil to our […]

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Novartis buys AVROBIO cystinosis gene therapy program for $87.5M

Jim Cornall — Fri, 26 May 2023 08:10:00 +0000

AVROBIO, Inc., a clinical-stage gene therapy company working to free people from a lifetime of genetic disease, has agreed to sell its investigational hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis to Novartis for $87.5 million in cash. AVROBIO retains full rights to its portfolio of first-in-class HSC gene therapies for […]

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Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

Jim Cornall — Fri, 26 May 2023 07:50:00 +0000

Rocket Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the company’s investigational lentiviral-based gene therapy for pyruvate kinase deficiency (PKD). PKD is a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. RMAT designation was granted based […]

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FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

Jim Cornall — Mon, 22 May 2023 15:23:26 +0000

The U.S. Food and Drug Administration has approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. The approval of Vyjuvek was granted to […]

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Can cardiovascular diseases be cured? The future of heart treatment

Willow Shah-Neville — Fri, 19 May 2023 14:01:04 +0000

Cardiovascular diseases, characterized as a group of disorders of the heart and blood vessels, are the leading cause of death worldwide. In fact, in the U.S. alone, it is thought that one person dies every 33 seconds from cardiovascular disease. Although clearly a huge health problem throughout the world, developing cures for cardiovascular diseases is […]

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8 rare diseases selected for gene therapy trial portfolio

Jim Cornall — Thu, 18 May 2023 09:15:00 +0000

The Foundation for the National Institutes of Health (FNIH) says the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. This portfolio pioneers a novel approach to gene therapy development for rare diseases by demonstrating that manufacturing and testing standards can provide a streamlined approval […]

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Sania Therapeutics launches to advance neural circuit dysfunction medicines

Jim Cornall — Thu, 18 May 2023 08:17:59 +0000

Sania Therapeutics, which develops genetic medicines for neural circuit dysfunction, has launched with the unveiling of its platforms at the American Society of Gene & Cell Therapy (ASGCT) conference. Sania Therapeutics’ approach combines the evolution of adeno-associated viruses (AAV) in human neural circuits, the fundamental components of the nervous system, and tuneable conditionally-activated ion channels. […]

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Cell and gene therapy company NewBiologix exits stealth with $50M

Jim Cornall — Fri, 12 May 2023 10:28:50 +0000

NewBiologix SA has emerged from stealth as a technology innovation company developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell therapies. The company’s DNA-based platform is being developed to address key manufacturing challenges associated with the production of recombinant adeno-associated virus (rAAV) vectors used to […]

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Delivering on the need for vector innovation in genetic medicine

External Contributor — Wed, 10 May 2023 07:30:00 +0000

By Tuyen Ong, CEO Ring Therapeutics, CEO-Partner Flagship Pioneering Gene therapy is a life-changing class of medicine that can treat patients with once intractable diseases. While revolutionary, significant limitations remain for the vectors that carry them. Viruses are masters at delivering genetic material to the nucleus of the organisms they infect, and because of this, […]

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Cell rejuvenation for age-related diseases

External Contributor — Wed, 10 May 2023 07:00:00 +0000

By Sharon Rosenzweig-Lipson, CSO, Life Biosciences Cellular rejuvenation is quickly becoming a hot topic in the development of novel therapeutic modalities for a range of diseases. The leading approaches to cellular rejuvenation currently in development utilize epigenetic reprogramming to achieve this goal. Epigenetics is the study of how positive and negative lifestyle factors (exercise, smoking, […]

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