XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

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Gene Therapy FDA Approval Gene In-Vivo 98

Roots Analysis

FEBRUARY 27, 2024

How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The global genome editing market is anticipated to grow at a CAGR of 12.6%

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Genome Genomics Gene Gene Editing 40

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene In-Vivo Life Science 130

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications.

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Gene Editing Gene Gene Therapy Drugs 59

Drug Discovery World

MARCH 7, 2024

Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

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Gene Therapy RNA Engineer Gene 52

The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. Our focus is now on accelerating progress to help realise the potential for durable and curative cell and gene therapies. 18, 2021 09:00 UTC. A total of $15.4

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Gene Therapy Gene In-Vivo Genetics 40

Drug Discovery World

MAY 29, 2023

Gene delivery technologies company VectorBuilder has opened a new office in Edinburgh, hoping to bolster its presence in the United Kingdom. The new location is part of Edinburgh Technopole, a Pioneer Group in Midlothian Science Zone.

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Gene Therapy In-Vivo Gene Sales 52

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

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Gene Therapy Gene Manufacturing Medicine 52

The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. MUNICH, Germany, Jan.

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Gene Therapy Gene Development In-Vivo 52

Pharmaceutical Technology

MAY 18, 2023

KSQ will receive future payments of up to $510m on achieving all milestones during the agreement period, along with potential net sales royalties on any commercial product that is the result of the partnership. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy In-Vivo Gene Genetics 130

Pharmaceutical Technology

MARCH 24, 2023

Through this collaboration, which builds on Generation Bio’s non-viral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement.” Both companies will use the partnership for advancing in vivo immune cell targeting as a new class of genetic medicines.

Genetics 130

Genetics Medicine Development In-Vivo 130

Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

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In-Vivo FDA Approval Gene Gene Editing 80

Pharmaceutical Technology

SEPTEMBER 29, 2022

Sanofi will also make tiered royalty payments on net sales of products resulting from the collaboration in the future. Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies.

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Development In-Vivo Genome Genomics 130

Pharmaceutical Technology

AUGUST 17, 2022

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . Merck will also make royalty payments on any approved products developed out of the partnership.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Pharmaceutical Technology

SEPTEMBER 14, 2022

As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. Tumour-infiltrating lymphocyte (TIL) therapy has been investigated as a treatment modality for melanoma for almost 20 years. months versus 3.1

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Immune Response In-Vivo Production Manufacturing 246

BioTech 365

SEPTEMBER 2, 2020

(Nasdaq: SRNE, “Sorrento”) and SmartPharm Therapeutics, Inc. (“SmartPharm”) announced today that Sorrento has completed the acquisition of SmartPharm, a gene-encoded protein therapeutics company developing non-viral DNA and … Continue reading →

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DNA In-Vivo Protein Gene 40

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Gene Therapy Marketing Gene DNA 52

pharmaphorum

OCTOBER 8, 2021

The cell therapy field is showing no signs of slowing down, with huge investment and excitement around next generation products and their clinical validation in solid tumour indications. This year, we’ll be specifically focusing on next generation cell therapies and discussing: Novel in vivo delivery.

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In-Vivo Gene Manufacturing Marketing 52

Roots Analysis

JANUARY 14, 2024

Overview of Biologics Biologics refer to medicinal / therapeutic products that are either manufactured using living organisms or semi-synthesized from biological sources. This can be attributed to the fact that the production of biopharmaceuticals requires living expression systems. Like all drugs, biologics are regulated by the FDA.

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Contract Manufacturing Manufacturing Protein Bacteria 40

pharmaphorum

MAY 27, 2021

Life sciences tools company Esco Lifesciences Group has scored $200m in a series A and crossover round co-led by Novo Holdings and Vivo Capital. Funds will be used to expand the company’s presence across Asia and create an innovation hub in Boston for cell and gene therapy technologies.

Life Science 59

Life Science In-Vivo Manufacturing Gene Therapy 59

Drug Discovery World

JUNE 16, 2023

The International Society for Cellular Therapy (ISCT) is a global society of clinicians, regulators, technologists, and industry partners with a shared mission to drive the translation of all cell and gene (CGT) therapies for the benefit of patients worldwide. DDW’s Megan Thomas heard from attendees about key trends in the sector.

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Manufacturing In-Vivo Production Compliance 52

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Early-stage companies are often years, often a decade or longer, from a marketable product.

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In-Vivo Marketing Engineer Genetics 52

Drug Discovery World

JUNE 14, 2023

The International Society for Cellular Therapy (ISCT) is a global society of clinicians, regulators, technologists, and industry partners with a shared mission to drive the translation of all cell and gene (CGT) therapies for the benefit of patients worldwide. Gene-modified MSC were presented as an example of MSC 2.0.

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In-Vivo Gene Manufacturing Development 52

Drug Discovery World

OCTOBER 27, 2023

With cues from a diverse population of tissue-specific cell types, extracellular matrix proteins, and biomechanical forces, cells grown in organ-chips closely emulate their in vivo counterparts and replicate tissue-level functions. But can organ-chips entirely replace other models? Not yet, according to Ewart.

Research 72

Research Gene Therapy In-Vivo In-Vitro 72

pharmaphorum

NOVEMBER 4, 2022

” At Novartis, she was involved with projects including multiple sclerosis therapy Kesimpta (ofatumumab), migraine therapy Aimovig (erenumab), and Parkinson’s disease, and the acquisition by Novartis of optogenetic gene therapy company Vedere Bio. .

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In-Vivo Gene Therapy Doctors Doctor 59

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.

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Business Development Development Gene Editing In-Vivo 40

XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level.

Gene Expression 112

Gene Expression Development RNA Drugs 112

Roots Analysis

SEPTEMBER 20, 2023

It is important to highlight that cell and gene therapies have been used to treat an array of medical conditions for which no other treatments are available. Further, the development of cell therapy products require a robust population of cells that can be subjected to genetic alterations, without being damaged in this process.

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Manufacturing Production Contamination Gene Therapy 40

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

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Gene Gene Therapy Gene Expression Regulation 40

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Lilly will provide funding for the Phase I clinical trials.

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Gene Editing Gene Gene Therapy In-Vivo 130

pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

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In-Vivo Gene Editing Trials Drugs 100

Pharmaceutical Technology

JULY 20, 2022

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes. Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes. Avista is entitled to receive $7.5m

Gene Therapy 147

Gene Therapy Gene Development In-Vivo 147

Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. The in vivo data showed statistically significant higher expression level and durability for circVec 2.1

RNA 52

RNA In-Vivo Gene Therapy Genetic Disease 52

Drug Discovery World

SEPTEMBER 14, 2023

Excluding Covid-19 medicines, total revenue increased 16% and product sales increased 15%. Business updates Pfizer reported second quarter 2023 revenues totalling $12.7 billion, a decrease of $15.0 billion, or 54%, compared to the prior year quarter, reflecting an operational decline of $14.7 Vaccines were up 9.1%

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Drugs Vaccination Vaccine Medicine 59

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

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In-Vivo In-Vitro Antibody Development 52