XTalks

NOVEMBER 26, 2024

The availability of a treatment that addresses both survival and quality of life represents a long-awaited milestone for patients and their caregivers. A Marketing Authorization Application has been submitted to the European Medicines Agency (EMA), with a decision anticipated in 2025.

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Genetic Disease Genetics Protein Clinical Development 105

Pharmaceutical Technology

FEBRUARY 2, 2023

The European Medicines Agency is currently reviewing a marketing authorisation application for daprodustat and a regulatory decision is expected in the first half of this year. Last December, GSK and Wave Life Sciences entered a strategic partnership to drive the discovery and development of oligonucleotide therapies for new genetic targets.

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FDA Approval Life Science Genetics Trials 264

pharmaphorum

NOVEMBER 7, 2022

This month, we highlight new appointments from Lumeris, Lineage Cell Therapeutics, 1910 Genetics, and ADC Therapeutics. . “Jill is a wonderful addition to our executive team as we work to establish Lineage as a leader in cell therapy and cell transplant medicine,” stated Brian M Culley, Lineage CEO. Ben Grabski, Lumeris.

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Life Science Genetics Business Development Marketing 105

XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. Here is a look at some life science trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.

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Life Science RNA Vaccination Vaccine 98

Pharmaceutical Technology

MAY 24, 2023

The BGTC is part of the accelerating medicines partnership (AMP) programme and is managed by the Foundation for the National Institutes of Health (FNIH). Streamlining the regulatory process, the AMP BGTC will also facilitate cost-efficient vector production to increase access for patients with rare and ultra-rare genetic diseases.

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Gene Therapy Gene Development Manufacturing 242

XTalks

DECEMBER 4, 2024

In the Asia-Pacific market, Jupiter has negotiated partnerships with companies such as Sichuan Kelun and Tianjin Pharmaceuticals, focusing on Jotrol’s integration into traditional Chinese medicine frameworks. International patents valid until 2036 further strengthen its global positioning.

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Bioavailability Genetic Disease Marketing RNA 111

XTalks

MAY 23, 2024

One of the more intriguing developments in cancer research in recent years is the growing understanding of clonal hematopoiesis, a phenomenon where blood cells expand from a single clone due to genetic mutations. Clonal hematopoiesis increases in prevalence with age and can precede hematologic malignancies.

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Genetics Research Genomics Genome 114

World of DTC Marketing

JANUARY 24, 2022

Particularly attractive are medicines ahead of the curve, aimed at diseases likely to move into the spotlight over the next few years. A group of early-stage life science investors and biotech CEOs joined together with a prominent patient advocate to speak out against H.R.3, Only about $2.2

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Life Science Drugs Development Genetic Disease 225

BioSpace

JUNE 13, 2024

Morgan’s life sciences venture capital arm on Thursday announced it closed its first biotech fund, targeting companies in cardiometabolic disease, immunology, oncology and genetic medicine.

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Life Science Genetics Medicine 83

pharmaphorum

OCTOBER 12, 2022

Now, he leads Whitelab Genomics as its artificial intelligence (AI) platform powers the development of genomic therapies – an emerging field in which genetic sequences are injected into cells to target and repair damaged genes. The evolving field of genomic medicine. This is just the beginning; the potential is enormous,” he says.

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Genome Genomics Medicine Development 105

XTalks

FEBRUARY 4, 2025

The company plans to use these funds to advance its groundbreaking research in small molecule precision medicines, targeting renal, cardiovascular and metabolic diseases, including obesity. For complex diseases like chronic kidney disease (CKD), where genetics play a significant role, such a platform holds promise.

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Clinical Trials Clinical Trials Trials Genetics 66

Pharmaceutical Technology

MAY 4, 2023

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration.

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Gene Therapy Gene Development Manufacturing 256

BioPharma Reporter

DECEMBER 16, 2022

GSK and genetic medicines company, Wave Life Sciences, have announced a strategic collaboration to advance oligonucleotide therapeutics across multiple disease areas.

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Life Science Genetics Development Medicine 52

XTalks

APRIL 13, 2022

Gentamicin is the preferred treatment for newborns with infections like sepsis but about one in 500 babies have a genetic variant that makes them susceptible to gentamicin-induced hearing loss. Genedrive’s Genetic Test Can Prevent Hearing Loss in Babies Through Personalized Treatment.

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Life Science Genetics Research Development 52

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. According to the FDA, life expectancy for those with DMD has increased over the years, with some patients surviving beyond 30 years.

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FDA Approval Genetics Gene Therapy Gene 105

XTalks

DECEMBER 20, 2023

In 2023, there were significant advancements and notable trends in the life sciences. Xtalks compiled a list of its top life science news and trends of 2023, which provided readers with the latest developments, information and expert insights across life science industries, including pharma, biotech and medical device.

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Life Science Clinical Trials Clinical Trials FDA Approval 59

XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

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Life Science Gene Therapy Gene Manufacturing 52

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

Genome 98

Genome Genomics Medicine Genetic Disease 98

Pharmaceutical Technology

MAY 22, 2023

“With this expansion we are strategically prepared, more than ever, to respond to our customers’ needs by supporting the accelerated delivery of critical vaccines and medicines to patients.” The bioprocess design centre offers expertise in bioprocessing and process scale-up.

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Genetic Analysis Manufacturing Drugs Vaccine 147

pharmaphorum

OCTOBER 2, 2020

Swiss medical data specialist Sophia Genetics has raised $110 million in an oversubscribed funding round that will be used to boost its headcount and international presence and prepare to take its shares public. The post $110m financing sets up US, Asia expansion for Sophia Genetics appeared first on.

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Genetics Genomics Genome Life Science 59

BioTech 365

SEPTEMBER 10, 2020

10, 2020 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, announced today that Paul Bolno, MD, MBA, President and Chief Executive … Continue reading →

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Life Science Genetics Medicine 40

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Solid Biosciences stated that the partnership boosts its scientific capabilities along with commercial potential in cardiac therapy.

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RNA Gene Therapy Genetics Genetic Disease 130

XTalks

NOVEMBER 15, 2024

Drug resistance in DEEs often stems from genetic and molecular factors unique to each syndrome, limiting standard treatments’ effectiveness. Bexicaserin’s development has also gained regulatory support in Europe, where the European Medicines Agency’s (EMA) Paediatric Committee endorsed its potential use in children as young as two.

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Trials Clinical Trials Clinical Trials Genetics 97

XTalks

JANUARY 20, 2025

From Bristol-Myers Squibbs monumental acquisition of Celgene to Pfizers recent purchase of Seagen, these deals illustrate the strategies and ambitions driving innovation in life sciences. The integration of Shire strengthened Takedas R&D pipeline, increasing its focus on innovative medicines and transformative therapies.

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Marketing Botox Life Science Pharma Companies 104

BioTech 365

SEPTEMBER 18, 2020

18, 2020 (GLOBE NEWSWIRE) — Homology Medicines, Inc. Nasdaq: FIXX), a genetic medicines company, announced today participation and presentations at the following virtual conferences: Oppenheimer Fall Healthcare Life Sciences & MedTech Summito Fireside Chat: September 22 at … Continue reading →

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Medicine Life Science Genetics 40

pharmaphorum

OCTOBER 7, 2020

There is enormous power in this genetic tool, which affects us all. It has not only revolutionised basic science, but also resulted in innovative crops and will lead to ground-breaking new medical treatments,” said Claes Gustafsson, chair of the Nobel Committee for Chemistry. What started as a curiosity?driven,

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Gene Editing Bacteria DNA Gene 105

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

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RNA Genetics Genetic Disease Medicine 105

pharmaphorum

OCTOBER 4, 2022

The life sciences company Solid Biosciences is merging with the privately-held gene therapy company AavantiBio in a deal which is expected to result in a combined sum of $215 million in cash and investments – projected to be enough for funding into 2025. This is due to close concurrently with the merger.

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Gene Therapy Gene Life Science Genetics 115

Pharmaceutical Technology

MAY 24, 2023

The ability to modify or introduce genetic material in human cells in such a precise and patient-centered manner clearly constitutes a breakthrough in personalized medicine. CGT drugs fall into two major categories: autologous and allogeneic.

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

XTalks

FEBRUARY 7, 2025

The clinical-stage biopharmaceutical company focuses on developing precision medicines for chronic kidney disease (CKD), cardiovascular and metabolic conditions. Utilizing its proprietary Compass platform, Maze analyzes genetic data to create targeted therapies that address the root causes of disease.

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Marketing Genetics Vaccination Vaccine 66

XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. Of the more than 10,000 rare diseases, more than 80 percent are caused by genetic defects.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

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. The company now plans to move this approach into a Phase III trial. PharmaTher is not the only player in this space.

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Research Trials Drugs Generic Drugs 275

pharmaphorum

DECEMBER 14, 2022

As many strive to bring about transformation in healthcare, Angelini Ventures takes a new approach to investments in digital health and life sciences companies that drive healthcare innovation and improve patient experiences. It aims to shape companies in the digital healthcare, connected medical devices, and life sciences spaces.

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Life Science Development Business Development Genetics 105

pharmaphorum

OCTOBER 7, 2021

However, developments in genomic medicine are making huge strides to improve this situation, and these will also be discussed during the webinar. He also serves as haematology consultant to the NICU and genetics service and is co-director of the NIH/Johns Hopkins Pediatric Hematology/Oncology Fellowship Program. About IQVIA.

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Genome Genomics Medicine Life Science 98

XTalks

FEBRUARY 25, 2025

CTX is a rare, progressive genetic disorder caused by mutations in the CYP27A1 gene, which disrupts the livers ability to produce chenodeoxycholic acid, a bile acid.

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FDA Approval Gene Therapy Gene Genetics 59

Scienmag

SEPTEMBER 22, 2020

– A Purdue Foundry-affiliated company with a growing reputation as a search engine for genome sequence data in life sciences has joined a nationally ranked health care startup accelerator. Credit: Karyosoft/Rajesh Perianayagam WEST LAFAYETTE, Ind.

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Genome Genomics Life Science Engineer 98

XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. Last year’s Nobel Prize in Physiology and Medicine also went to RNA researchers.

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Regulation Genetics Scientist RNA 52