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.

Gene Therapy 242

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.

Bioavailability 111

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.

Genetics 114

Genetics Research Genome Genomics 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

Life Science 225

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.

Life Science 83

Life Science Genetics Medicine 83

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.

Clinical Trials 66

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.

Gene Therapy 256

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.

Life Science 52

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.

Life Science 52

Life Science Genetics Research Development 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

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.

Genetics 59

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 →

Life Science 40

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.

RNA 130

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.

Trials 97

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.

Marketing 104

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 →

Medicine 40

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,

Gene Editing 105

Gene Editing Bacteria DNA Gene 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.

Gene Therapy 115

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.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

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.

Gene Therapy 98

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.

Research 275

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.

Life Science 105

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.

Genomics 98

Genomics Genome 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.

FDA Approval 59

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.

Genome 98

Genome Genomics Life Science Engineer 98

XTalks

JANUARY 26, 2023

Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations. Results from the ALPINE study have been published in the New England Journal of Medicine. percent vs 72.9

FDA Approval 97

FDA Approval Life Science Clinical Trials Clinical Trials 97

pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Furthermore, each cancer has various gene changes – also referred to as the genetic makeup of cancer. Tailored oncology. Navigating barriers to access.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

pharmaphorum

MARCH 29, 2021

The way NICE reviews new medicines is about to go through its biggest change in years – but what do the proposals mean for the future of drug development in the UK? Part of this is about making sure the processes we use to evaluate new medicines are fit for purpose. Evolving landscape. Addressing uncertainty.

Medicine 93

Medicine Life Science Drugs Development 93

XTalks

MAY 26, 2023

DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.

Gene Therapy 97

Gene Therapy Gene Dermatology FDA Approval 97

Pharmaceutical Technology

JANUARY 22, 2023

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. Elsewhere, Albireo Pharma is aiming to file for supplemental NDA to the FDA for its drug Bylvay (odevixibat), which could be the second treatment for Alagille syndrome, a rare genetic disorder caused by a JAG1 gene mutation.

Drugs 317

Drugs Gene Hormones Clinical Trials 317

XTalks

JANUARY 26, 2024

By targeting and identifying specific genetic mutations, this technology could lead to the development of more precise diagnostic tools, enabling early intervention and treatment. Personalized medicine: One of the most exciting prospects of CRISPR/Cas9 in oncology is its contribution to personalized medicine.

Research 118

Research Gene Editing Gene Genetics 118

Scienmag

JUNE 7, 2021

Credit: Autobahn Labs Cold Spring Harbor Laboratory (CSHL) is collaborating with Autobahn Labs, a new life sciences incubator, to catalyze the Laboratory’s early-stage discovery programs into spin-out companies that commercialize transformational new therapies. Autobahn will provide […].

Genetic Disease 52

Genetic Disease Life Science Genetics Research 52

XTalks

MAY 31, 2022

This investment will strengthen Eli Lilly’s manufacturing capacities towards treatments, specifically active ingredients, and genetic medicines. The company previously announced that their novel medicines are on track to become the next generation of treatments for several diseases including diabetes, obesity and more.

Manufacturing 98

Manufacturing Medicine Genetics Drugs 98

XTalks

MAY 20, 2022

Depending on the type of field or company, you can pursue clinical scientist jobs with a major in biology, medicine, medical technology, chemistry, immunology and other related life science and pre-medical degrees. Clinical Pharmaceutical Science. Genetic Science. Computer Science and Modeling.

Scientist 105

Scientist Life Science Clinical Research Doctors 105

pharmaphorum

AUGUST 11, 2022

The participants undergo monitoring for genetic variations that could potentially negatively impact their reaction to therapies. The best way to do that is to target specific sub-populations, which will serve as a natural lead into personalised medicine becoming more mainstream. About the Author.

Genome 98

Genome Genomics Drugs Genetics 98

XTalks

SEPTEMBER 18, 2024

Alnylam is focused on developing RNAi medicines to transform the way diseases like cardiovascular and neurological diseases are treated. Brown discusses the promise and evolving landscape of RNAi therapeutics across various therapeutic areas and Alnylam’s approach to developing innovative RNAi medicines.

Life Science 52

Life Science Research Medicine Development 52

XTalks

SEPTEMBER 28, 2020

The leading global life science company is set to introduce Resolution Bio’s ctDx Lung assay , a non-invasive liquid biopsy test for patients with non-small cell lung cancer (NSCLC). Based on the genetic biomarker(s) identified, the most effective therapy targeting the specific molecular marker can be selected.

Gene 105

Gene DNA Life Science Genotype 105