Drug Discovery World

NOVEMBER 24, 2022

With European life sciences R&D investment at a 20-year low, biopharmaceutical associations and MEPs are calling on the EU to modernise its policies. Fortunately, the EFPIA report also offers a detailed list of recommendations and the hope that with a proactive approach to nurturing life sciences, this trend can be reversed. .

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XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). The FDA approval came after several delays and a narrow advisory committee vote of eight to six in favor of the gene therapy’s risk-benefit profile.

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Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

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BioPharma Reporter

AUGUST 14, 2023

Teknova, a producer of life science reagents, has opened a new GMP-certified production facility which it claims will enable bioprocessing and gene therapy companies to get into the clinic faster.

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BioPharma Reporter

JULY 3, 2023

Donaldson, a provider of filtration products and solutions, has acquired Univercells Technologies, a producer of biomanufacturing solutions for cell and gene therapy research, development, and commercial manufacturing, to broaden its life sciences reach.

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Drug Discovery World

FEBRUARY 12, 2024

In the upcoming year, we will see the expansion of new, more complex markets and updated requirements from regulators. Ensuring sufficient and timely patient enrollment in a rapidly growing number of cell and gene therapy clinical trials is becoming a bottleneck to advancing potentially life-saving therapies.

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BioPharma Reporter

MARCH 22, 2022

Fujifilm Irvine Scientific, Inc, has announced the acquisition of Shenandoah Biotechnology, a company supplying recombinant proteins to the drug discovery, life science research, and cell and gene therapy markets.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2. Conclusion.

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Drug Discovery World

OCTOBER 24, 2023

While attending Life Science Baltics 2023, Diana Spencer spoke to Monika Paule , CEO and Co-founder of Caszyme, about creating a company based on CRISPR gene editing and some of the challenges of operating in this dynamic area of research. Caszyme was founded in 2017 by the pioneers of CRISPR gene editing technologies.

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Drug Discovery World

MAY 6, 2024

Thanks to a prestigious academic legacy, Europe has long been a dominant region for life sciences. Europe is traditionally a life sciences powerhouse, with a strong tradition in pharmaceutical discovery. The oldest, still active pharmaceutical company worldwide, Merck, started life in 1668 in Darmstadt, Germany.

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Drug Discovery World

JULY 18, 2023

Chris Hughes, Managing Director North Europe, Revvity, said: “We are thrilled to be part of the UK’s life sciences industry through our Scientific Centre of Excellence and top expertise in Cambridge. The centre also features a newly built demo lab which will bring the Revvity portfolio closer to scientists.

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Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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The Pharma Data

OCTOBER 26, 2020

The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Sensorion is currently advancing two preclinical gene therapy programs conducted under a broad multi-program research partnership with the Genetics and Physiology of Hearing Unit at Institut Pasteur (Paris).

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Gene Therapy Gene Manufacturing Production 52

Drug Discovery World

FEBRUARY 19, 2024

Financial landscape In 2023, life science companies experienced a significant decrease in funding, particularly seed funding for early-stage drug development. The transformative impact of gene therapy, particularly for paediatric and neurodegenerative conditions, cannot be overstated.

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Development Gene Therapy Drugs Antibody 59

XTalks

MARCH 13, 2024

Related: Transforming Eye Care: Innovating New Glaucoma and Dry Eye Disease Treatments — Featuring Pierre Simon, VP Marketing, Sight Sciences – Xtalks Life Science Podcast Ep. There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease.

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Drug Discovery World

NOVEMBER 27, 2023

By Anis H Khimani , PhD, Senior Strategy Leader, Life Sciences strategy group, Revvity, Inc. In addition, it has stimulated increased collaboration among drug developers, technology providers, supply chain organisations, and regulators. link] About the author Anis is within the strategy group in Life Sciences at Revvity, Inc.

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Production Engineer Gene Editing Gene 59

ACRP blog

APRIL 5, 2023

The life sciences sector is increasingly leveraging technology across its operations, and this trend will continue in 2023 with more collaborations and partnerships between pharmaceutical, biotechnology, and medical device companies and information technology vendors, Lyons adds.

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pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. I encourage continued support of fundamental science as well as public discourse about the ethical uses and responsible regulation of CRISPR technology.”.

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XTalks

AUGUST 3, 2023

As a comparatively new discipline in the life sciences, leaders like Raphael Pareschi must ensure that they’re consistently engaged with the global pharmacovigilance community to maintain patient access to safe drugs. What are some of the future challenges you see in the approval of novel treatments, including cell and gene therapies?

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XTalks

OCTOBER 7, 2020

Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. Born with spinal muscular atrophy (SMA), the little boy was saved by the drug Zolgensma, a one-time intravenous infusion that restored the child’s defective SMN1 gene. Adjusting for COVID-19.

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Drug Discovery World

JUNE 15, 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. Regulation is necessary, but optimising timescales would be ideal.

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Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

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XTalks

MARCH 28, 2024

Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein. It will also be going up against its breakthrough gene therapy Elevidys (delandistrogene moxeparvovec), which received FDA approval last year. The drug generated domestic sales of $200.4

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XTalks

JUNE 12, 2023

Moreover, Lonza, a global manufacturing partner for the pharmaceutical, biotech and nutrition industries, announced that two cell and gene therapies manufactured at their Houston site got FDA approval in 2022.

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XTalks

JANUARY 26, 2023

Brukinsa is a small-molecule inhibitor of Bruton tyrosine kinase (BTK), which is a tyrosine kinase encoded by the BTK gene. BTK plays an important role in the immune system by regulating B cell receptor signaling through its effects on the development and maturation of B cells.

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XTalks

MARCH 25, 2021

Xtalks is proud to announce the launch of the Xtalks Life Science podcast. Subscribe to the Xtalks Life Science Podcast to never miss a new episode. Fresh Conversations About Life Science Topics. Diverse Voices for Lively Discussions. Sarah is also an experienced webinar moderator.

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XTalks

DECEMBER 12, 2023

PNH is caused by a mutation in the CFB gene that codes for complement factor B, a component of the alternative pathway of complement activation. Mutations in the gene result in the production of red blood cells that are prone to premature destruction (hemolysis).

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Pharmaceutical Technology

JANUARY 31, 2023

The US omnibus bill passed in late December 2022 will give the agency greater powers to regulate accelerated approval drugs, while the Inflation Reduction Act’s lowering of drug prices will create an uncertain environment for drug developers, he said. It’s much needed. It’s a hot area.” trillion Consolidated Appropriations Act.

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Drug Discovery World

SEPTEMBER 12, 2022

Chaired by Karin Blumer, Director of Global Patient Engagement at Novartis, the first event in this track, called ‘Patient Engagement: Driving Patient Advocacy to Inform Patient Centric Development’, looked into the multi-layered importance of involving patients in the drug development process, particularly in this case for gene therapy.

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Clinical Trials Clinical Trials Trials Gene Therapy 52

XTalks

OCTOBER 18, 2022

Read on to learn why life science and clinical research professionals are choosing a career with Medpace. Being a scientifically-driven and therapeutically aligned CRO, candidates can put their life-science backgrounds to work in a broad range of positions across a range of therapeutic and specialty areas. Nephrology.

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Clinical Research Research Clinical Trials Clinical Trials 111

Drug Discovery World

JULY 11, 2023

Tim Lowery , President, JSR Life Sciences, asks whether artificial intelligence can do for life sciences what it has done in other sectors and whether these tools can keep up with the complexities of human biology. So, where do we go from here to realise their potential in life sciences?

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Drug Discovery World

AUGUST 1, 2023

Evonetix will use the funding to brings its semiconductor chips to commercial scale and extend gene assembly capabilities for its binary assembly technology to deliver accurate, gene-length DNA in a benchtop instrument. Evonetix is in a very exciting phase, having recently opened our early access program.

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The Pharma Data

JANUARY 27, 2021

FDA Also Grants Two Orphan Drug Designations for the Company’s Gene Therapy Programs for Gaucher Disease and Mucolipidosis –. Food and Drug Administration (FDA) granted six rare pediatric disease designations (RPDDs) for various programs within its development pipeline for LSDs, including four recombinant enzyme and two gene therapy programs.

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Drug Discovery World

APRIL 11, 2023

Read more: Synthesised sea sponge molecule could fight Parkinson’s Changing the therapeutic focus Researchers identified that a gene associated with an increased risk of Parkinson’s Disease also contributes to a build-up of cell debris in the brain. USP30 inhibition has been proposed as a therapeutic strategy for Parkinson’s disease.

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XTalks

JANUARY 17, 2024

Changes in what clinical performance data is used to inform the trial design and site selection, new approaches to patient recruitment and the rise of advanced treatments such as cell and gene therapy are rewriting the script. It’s very positive and it’s going to help move forward the pathway for new drugs.”

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Cloudbyz

JUNE 10, 2023

21 CFR Part 11 is a crucial component of regulatory compliance in clinical trials and, more broadly, any industry subject to the regulations of the U.S. The regulation was established in 1997 to address the FDA’s concerns regarding the use of electronic records and signatures, ensuring their integrity, accuracy, and confidentiality.

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