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.

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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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XTalks

DECEMBER 28, 2023

XTALKS WEBINAR: Impacts of the New IVD Regulation (IVDR) for Manufacturers and Users Live and On-Demand: Tuesday, January 9, 2024, at 10am EST (4pm CET/EU-Central) Register for this free webinar to learn how the new In Vitro Diagnostic Regulation (IVDR) impacts manufacturers and users of diagnostic devices.

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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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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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XTalks

MARCH 25, 2021

Sydney writes about various food industry topics, including safety and regulation, grocery and foodservice, innovation and sustainability. She focuses on news relating to the food industry and writes blogs on recruitment and HR in the life sciences. Mira Nabulsi, MJ , is a multimedia editor and webinar moderator at Xtalks.

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XTalks

MAY 17, 2024

For years, GLP-1 receptor agonists have been at the forefront of treating conditions like type 2 diabetes and obesity, celebrated for their dual action in regulating blood sugar and aiding weight loss. Yet, it’s not the past successes that captivate me the most—it’s the untapped potential of precision medicine in this field.

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XTalks

APRIL 3, 2024

Related: Novartis’ Fabhalta Gets FDA Approval for Rare Complement Blood Disorder The FDA’s green light for Voydeya comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator in the world to back the therapy. AstraZeneca is also venturing into cell therapy and genetic medicine.

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XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. 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. One chapter discusses how to form early partnerships with regulators.

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pharmaphorum

JUNE 20, 2022

Evaluate provides commercial intelligence to the pharma and life sciences sectors through annual publications. There have been continued calls to reform orphan drug legislation in the US and the EU, particularly the 40-year-old Orphan Drug Act and the European Union’s 1999 Orphan Drug Regulation. About the author.

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pharmaphorum

JANUARY 12, 2022

EMA “lags behind” other international regulators when it comes to approval timelines, finds EFPIA analysis. Only a regulatory framework that “embraces a culture of excellence and innovation” will make Europe a world leader in life sciences and ensure people can reap the benefits of life-changing new treatments.

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XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

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XTalks

SEPTEMBER 12, 2022

ASMD is a rare genetic lysosomal storage disease caused by mutations in the sphingomyelin phophodiesterase-1 ( SMPD1 ) gene that codes for the acid sphingomyelinase (ASM) enzyme. ASM is required for breaking down a complex lipid called sphingomyelin. Related: FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures.

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pharmaphorum

DECEMBER 10, 2020

For more than thirty years Niall Dickson has been at the centre of health policy and regulation and most recently was CEO of the NHS Confederation, representing UK health service organisations. Grace’s academic background is in biology specialising in genetics.

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XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Early and ongoing engagement with regulators greatly helps clinical development planning. As a result, there are several EU regulations and directives to follow.

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Pharmaceutical Technology

NOVEMBER 29, 2022

PharmaTher also has an agreement in place with Case Western Reserve University to develop ketamine as a treatment for Rett syndrome, a rare genetic neurological disorder. In March 2019, the American regulator approved esketamine , an enantiomer of ketamine, for use in treatment-resistant depression as a nasal spray.

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Drug Discovery World

MAY 25, 2023

With over 150 pharma services/supply companies and 19 universities, Scotland is one of the most advanced life science sectors in the UK and the world. Using complex models of cancer for preclinical testing After a coffee break, Prof Sansom was back behind the podium talking about the MRC National Mouse Genetics Network.

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XTalks

JUNE 28, 2023

DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial.

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XTalks

SEPTEMBER 17, 2021

This has brought criticism that US regulators are moving too slow on a drug that has clinical promise and is being backed by other international regulators. The European Medicines Agency (EMA) is also pushing ahead on the ALS drug candidate.

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Drug Discovery World

FEBRUARY 6, 2024

However, many have cautioned against too great a reliance on these tools and emphasised the importance of checks and balances to ensure the systems are properly regulated and results are verified. By making lab automation as easy as using a smartphone, the Flex robot democratises access to automation in life sciences research.”

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pharmaphorum

MARCH 4, 2021

Stringent regulations in healthcare mean that the sector often fares better in data bias compared to what Rahman terms the ‘Wild West’ of AI in more general settings – but there is also more potential for harm if healthcare gets this wrong. Abid leads the development of Cognitive Core, an AI platform created for the life sciences industry.

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The Pharma Data

OCTOBER 26, 2020

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). Novasep provides cost-effective and sustainable manufacturing solutions for the life sciences industries. About Novasep.

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pharmaphorum

DECEMBER 16, 2022

There are already rules that dictate how countries should react against public health events that have the potential to traverse country borders, through the International Health Regulations. In response, some companies, such as Pfizer , have made commitments to sell products to the countries at non-profit prices.

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The Pharma Data

JANUARY 27, 2021

–( BUSINESS WIRE )– M6P Therapeutics , a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced that the U.S.

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XTalks

MARCH 10, 2023

Previous animal model studies of Friedreich’s ataxia showed a dysfunction in the regulation of Nrf2 signaling, a pathway associated with reduced inflammatory response and improved mitochondrial function. Another study showed that omaveloxolone restored mitochondrial function in fibroblasts from Friedreich’s ataxia patients.

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Drug Discovery World

AUGUST 3, 2023

Medical cannabis is often a first-thought when regarding plant-based medicines, but there remains public scepticism, arguably due to the regulations relating to the substance. My mission is to see cannabis regulators understand basic science and to consult experienced scientists to refine regulations.

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pharmaphorum

JUNE 11, 2021

. “Cyber attacks within the healthcare and pharmaceutical industries have become more frequent and damaging” Clinical trial records often include very detailed information about individuals: habits and genetic information about a patient or their immune system. About the author.

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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

APRIL 5, 2021

Having a strong background in biochemistry or genetics is necessary for the problem-solving element of the job. Furthermore, the necessary knowledge and understanding of research requirements, regulations and protocols to comply with all federal rules is an asset. Bioinformatics Engineer. Job Description.

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pharmaphorum

DECEMBER 22, 2021

The NICE methods and process review, he said, represents an opportunity to create a modern, world-class appraisals system that “fosters innovation and shows the global life sciences sector that UK plc is open for business”. Under EU regulation No. Patient involvement is central to this, he said.

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XTalks

FEBRUARY 24, 2023

When life sciences industry partners with patients and their advocacy communities, the patient voice becomes integrated into the drug development process. Many rare disease communities are seeing gene therapies as potential game changers, since most rare diseases are genetically driven,” Dr. Raymond says.

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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 primary drivers for growth include increasing cancer and genetic cases and predicated applicability of CGT as a treatment.

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The Pharma Data

DECEMBER 17, 2020

Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. Immune Regulation – U.K.-based based Immune Regulation Ltd. He spent 27 years at Merck & Co.

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XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

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XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. Defects in hamartin or tuberin lead to a loss of regulation of mTOR, resulting in aberrant differentiation and development, which causes the generation of enlarged cells, as seen in TSC brain lesions.

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XTalks

SEPTEMBER 17, 2020

Often, the potential patient pool for a given trial is small and the unmet need is high, incentivizing drugmakers and regulators to find innovative ways to make game-changing therapeutics available to those who need them most. Duchenne Muscular Dystrophy. Prevalence: 1 in 3,500. Lipodystrophy. Prevalence: 1 in 10 million.

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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. As the field matures and the risks are better understood, regulators continue to streamline duplicative and burdensome oversight efforts 5.

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