Drugs and Genetics - Clinical Research Informer

World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing

Worldwide Clinical Trials

DECEMBER 19, 2024

At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.

Genetics

Genetics In-Vitro Drugs Clinical Trials

FDA clears AstraZeneca, Merck drug to treat some genetic breast cancers early

Bio Pharma Dive

MARCH 14, 2022

Lynparza is the first drug targeting BRCA mutations to be approved for use in early breast cancer, a decision that could lead to more genetic testing.

Genetics

Genetics Drugs

Merck pays Seattle Genetics $1.7B as antibody cancer drugs continue comeback

Bio Pharma Dive

SEPTEMBER 14, 2020

Merck's deal for Seattle Genetics' antibody-drug conjugate comes as Gilead agreed to acquire rival developer Immunomedics in the biotech industry's largest buyout of 2020.

Genetics

Genetics Antibody Drugs Development

Webinars

Bridging Innovation & Patient Care: The Growing Role of AI

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FDA approves Regeneron drug for rare, genetic form of high cholesterol

Bio Pharma Dive

FEBRUARY 12, 2021

The biotech set an average annual price of $450,000 for the first-of-its-kind drug, which treats a condition that affects about 1,300 people in the U.S.

FDA Approval

FDA Approval Genetics Drugs

An AstraZeneca, Merck drug slows the return of genetic breast cancer. Will testing speed up?

Bio Pharma Dive

JUNE 3, 2021

Study results disclosed ahead of ASCO could make Lynparza a standard "adjuvant" treatment for people with an inherited form of breast cancer — as long as they know they have it.

Genetics

Genetics Drugs

Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

DECEMBER 18, 2024

Neurocrine Biosciences Crenessity (crinecerfont) has been given the green light by the US Food and Drug Administration (FDA) as a new treatment for congenital adrenal hyperplasia (CAH). CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens.

Genetics

Genetics Hormones Trials Drugs

FDA clears first-of-its-kind Duchenne drug for broad use

Bio Pharma Dive

MARCH 22, 2024

The approval of Italfarmaco’s Duvyzat is the first for an oral, non-steroidal Duchenne drug and was issued for all patients at least six years of age, regardless of their genetic underpinnings.

Drugs

Drugs Genetics

Genetic diagnostics promises paradigm shift towards personalized, proactive healthcare

AuroBlog - Aurous Healthcare Clinical Trials blog

FEBRUARY 18, 2024

Indian healthcare industry is of the view that emergence of genetic diagnostics will be a paradigm shift towards personalized medicine. Through the ever-evolving landscape of healthcare, the role of genetic diagnostics offers personalized insights and proactive solutions for disease management and prevention.

Genetics

Genetics Medicine Development Drugs

Ovid turns to gene therapy startup to restock drug pipeline

Bio Pharma Dive

AUGUST 24, 2022

The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.

Gene Therapy

Gene Therapy Bacteria Gene Drugs

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

Genetics

Genetics Trials Clinical Trials Clinical Trials

Amgen to test new way to lower heart risk with large drug trial

Bio Pharma Dive

NOVEMBER 7, 2022

Buoyed by strong mid-stage study results, the biotech will soon start a cardiovascular outcomes trial of a drug designed to target a genetic risk factor called lipoprotein(a).

Drug Trials

Drug Trials Trials Genetics Drugs

Seattle Genetics stays step ahead of rivals with bladder cancer data

Bio Pharma Dive

SEPTEMBER 18, 2020

Data confirming the company's drug Padcev can extend the lives of bladder cancer patients should strengthen its position with regulators, although Immunomedics is close behind with a competing treatment.

Genetics

Genetics Regulation Drugs

Merck wins approval for cancer drug acquired in 2019 biotech buyout

Bio Pharma Dive

AUGUST 16, 2021

The FDA cleared Welireg, which Merck picked up in its $1 billion takeover of Peloton Therapeutics, to treat certain tumors associated with a rare genetic disease.

Genetic Disease

Genetic Disease Genetics Drugs

Indian healthcare looks at pharmacogenomics in patient care as comprehensive genetic tests gain ground

AuroBlog - Aurous Healthcare Clinical Trials blog

NOVEMBER 14, 2023

Indian healthcare looks at pharmacogenomics, the study of influence of genetic factors on drug response, in patient care as comprehensive genetic tests gain ground.

Genetics

Genetics Drugs Clinical Trials Clinical Trials

Ionis, AstraZeneca change a key trial, fueling more questions about a genetic heart disease

Bio Pharma Dive

MAY 2, 2022

The partners increased the size and length of a large, ongoing study in transthyretin amyloidosis cardiomyopathy, a decision that has implications Alnylam Pharmaceuticals, which is testing two of its drugs against the disease.

Genetics

Genetics Trials Drugs

On the hunt for new ALS drugs, researchers see progress, and a long road ahead

Bio Pharma Dive

DECEMBER 12, 2021

But despite continued setbacks, drug developers are hopeful that genetic technologies and a better understanding of biology will bring new options in the not-too-distant future The fatal nerve disease has few treatments.

Genetics

Genetics Drugs Research Development

Regeneron, in search of an eye gene therapy, turns to a young biotech

Bio Pharma Dive

APRIL 6, 2022

Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.

Gene Therapy

Gene Therapy Gene Genetics Medicine

STAT+: Emerging class of genetically targeted drugs induce remissions in leukemia patients

STAT News

DECEMBER 10, 2022

NEW ORLEANS — An emerging class of genetically targeted drugs is inducing remissions in about one-third of patients with advanced leukemia, according to updates Saturday from separate clinical trials. Continue to STAT+ to read the full story…

Genetics

Genetics Drugs Clinical Trials Clinical Trials

SOPHiA GENETICS and AstraZeneca partner for oncology drugs development

Pharmaceutical Technology

FEBRUARY 13, 2023

SOPHiA GENETICS is expanding its collaboration with AstraZeneca to include multimodal approaches for developing cancer drugs. This collaboration aims to use the global SOPHiA DDM platform, a cloud-native platform of SOPHiA GENETICS, and multimodal algorithmic capabilities for AstraZeneca’s oncology portfolio.

Genetics

Genetics Development Drugs Genome

UniQure moves Huntington's gene therapy to next phase of key trial

Bio Pharma Dive

MAY 27, 2021

Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the genetic disease.

Gene Therapy

Gene Therapy Genetic Disease Gene Genetics

Synlogic’s SYNB1934 receives orphan drug status from US FDA

Pharmaceutical Technology

MAY 10, 2023

Synlogic has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for SYNB1934 to treat phenylketonuria (PKU), a rare inherited metabolic disease. The orally administered, non-systemically absorbed drug candidate SYNB1934 has been designed for reducing blood phenylalanine (Phe) levels in PKU patients.

Genetic Engineering

Genetic Engineering Drugs Engineer Genetics

Gene therapy startup emerges with green light for first-of-its-kind trial

Bio Pharma Dive

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

Gene Therapy

Gene Therapy Gene Genetics Trials

UK agency pilots biobank to study links between genetics and drug side effects

Pharmaceutical Technology

MAY 25, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) aims to launch a pilot genetic biobank that will gather patient data to associate drug-related adverse events to their genetic makeup. According to the press release, the MHRA will be the first drug safety regulator in the world to pilot such a biobank.

Biobanking

Biobanking Genetics Genome Project Genome

Lilly adds to genetic meds push with Genetic Leap alliance

pharmaphorum

SEPTEMBER 6, 2024

Eli Lilly signs another genetic medicines deal, this time with Genetic Leap, a start-up that applies AI to the discovery of RNA-targeted drugs.

Genetics

Genetics RNA Medicine Drugs

Lilly gene therapy finding puts focus on hearing loss treatment pipeline

Bio Pharma Dive

JANUARY 24, 2024

An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. are taking aim at the same drug target. Others, including biotechs in China and Regeneron in the U.S.,

Gene Therapy

Gene Therapy Gene Genetics Medicine

Biogen shelves ALS drug after early-stage trial failure

Bio Pharma Dive

MARCH 28, 2022

The study setback raises questions over how well a type of genetic medicine can work in adults with central nervous system disorders like ALS.

Genetics

Genetics Trials Medicine Drugs

Mirum drug for rare genetic disease gains FDA approval

Bio Pharma Dive

FEBRUARY 24, 2025

Mirum gained the drug, which it will sell as Ctexli for adults with cerebrotendinous xanthomatosis, in a 2023 deal with Travere Therapeutics.

Genetic Disease

Genetic Disease FDA Approval Genetics Drugs

Fulgent Genetics acquires therapeutics development company Fulgent Pharma

Pharmaceutical Technology

NOVEMBER 8, 2022

Fulgent Genetics has acquired clinical-stage therapeutics development firm Fulgent Pharma for a total deal price of nearly $100m. According to the deal, the purchase price, contingent on adjustments, has to be paid by Fulgent Genetics as a combination of cash on hand and its shares of common stock.

Genetics

Genetics Development Genome Genomics

“Game-changing” obesity drug over-promising?

World of DTC Marketing

NOVEMBER 5, 2021

It’s being positioned as an anti-obesity drug, but one study by Novo Nordisk has shown that people who stop taking Wegovy after a few months tend to regain much of their lost weight within a year. Patients want quick fixes, and the idea of losing some weight because of a new drug is enticing to them. Nearly 3% of the overall U.S.

Drugs

Drugs Pharmacy Clinical Trials Clinical Trials

UCB enters drug discovery collaboration with Aitia

Pharmaceutical Technology

MARCH 16, 2023

Global biopharmaceutical firm UCB has entered an early drug discovery collaboration with Aitia. The collaboration is aimed at discovering and validating new drug targets and drug candidates that are linked to clinical endpoints causally in Huntington’s disease, a debilitating genetic disorder.

Drugs

Drugs Genetics Development Medicine

FDA grants orphan drug designation to DTx Pharma’s CMT1A therapeutic

Pharmaceutical Technology

JUNE 8, 2023

The US Food Drug Administration (FDA) has granted orphan drug designation to DTx Pharma’s investigational DTx-1252 for the treatment of Charcot-Marie-Tooth disease Type 1A (CMT1A). DTx-1252 targets the underlying genetic lesion of the disease and leverages our FALCON platform to unlock the promise of RNAi therapeutics. “We

Drugs

Drugs Gene Therapy Gene RNA

Indian pharma to take advantage of GenAI potential to streamline drug discovery, reduce cost & time-to-market

AuroBlog - Aurous Healthcare Clinical Trials blog

JULY 28, 2024

The Indian pharmaceutical industry is working to take advantage of Generative AI potential to transform drug discovery. Functions such as establishing connections between the causative pathways and showing the genetic predispositions have the potential to enable successful interventions and revert disease progression.

Genetics

Genetics Drugs Marketing Clinical Trials

Alkeus raises funds to launch Stargardt disease therapy gildeuretinol

Pharmaceutical Technology

JUNE 6, 2023

Alkeus Pharmaceuticals has raised $150m in Series B financing to support the registration and launch of gildeuretinol (ALK-001) to treat Stargardt disease, a genetic cause of blindness in children and young adults. Alkeus Pharmaceuticals intends to submit a new drug application for gildeuretinol in 2024.

Genetics

Genetics Life Science Trials Clinical Development

Stoke shares surge on updated results for Dravet drug

Bio Pharma Dive

MARCH 26, 2024

The biotech is trying to develop a Spinraza-like drug for the condition, a form of genetic epilepsy. Fresh data appear supportive of a high dose’s effectiveness.

Drugs

Drugs Genetics Development

Leading innovators in genetically modified animal models for the pharmaceutical industry

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Genetically modified animal models. Regeneron Pharmaceuticals is one of the leading patent filers in the generation of genetically modified animal models.

Genetics

Genetics DNA Genome Genomics

Mirai Bio launches with plan to help genetic med developers

pharmaphorum

SEPTEMBER 26, 2024

Flagship Pioneering's latest biotech launch, Mirai Bio, aims to help genetic medicine developers find the best way to get their drugs to patients.The new Cambridge, Massachusetts-based company – which was set up in 2021 but makes its official launch today with $50 million in funding from Flagship – applies artificial intelligence to solve the various (..)

Genetics

Genetics Development Medicine Drugs

Integrating Bioanalytical Automation, Biomarkers, Pharmacogenomics, & Cost-Effective Strategies

Worldwide Clinical Trials

JANUARY 6, 2025

Tom Zhang, PhD, Chief Scientific Officer, Large Molecule Bioanalysis Revolutionizing Therapeutic Development with Cutting-Edge Bioanalysis Automation Cutting-edge advancements in bioanalysis (BioA) and drug R&D are reshaping the future of preventative and interventional care, but how do we harness these to their fullest potential?

Clinical Trials

Clinical Trials Clinical Trials Trials Genetics

Pharmacognosy and Drug Patents: Unveiling Nature’s Medicinal Treasures

Drug Patent Watch

DECEMBER 9, 2024

This intersection is where pharmacognosy meets drug patents, creating a unique landscape that shapes the future of medicine. But what exactly is pharmacognosy, and how does it relate to the complex world of drug patents? This approach has led to the discovery of numerous potential drug candidates. What is a Drug Patent?

Medicine

Medicine Drugs Production Development

Gene Therapy and Pharmacokinetics

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing. Gene Therapy Definition.

Gene Therapy

Gene Therapy Gene Gene Editing Genetics

BridgeBio data show sustained benefit for achondroplasia drug

Bio Pharma Dive

JUNE 4, 2024

Longer follow-up results from a mid-stage trial indicate the company’s treatment for a genetic cause of dwarfism may be competitive to an approved therapy from BioMarin Pharmaceutical.

Genetics

Genetics Drugs Trials

Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

XTalks

JANUARY 6, 2025

The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.

Gene Therapy

Gene Therapy Protein Genetic Disease Gene

No drugs for prions: could new approaches alleviate therapeutic scarcity?

Pharmaceutical Technology

AUGUST 5, 2022

No progress in drug discovery. “We On the other hand, I think the progress in drug discovery is nil,” comments Requena on the prion disease landscape. According to GlobalData’s Pharma Intelligence Center, there are only six drugs in active development for CJD, of which only one has been in clinical trials.

Drugs

Drugs Protein Vaccine Vaccination

FDA widens use of cancer drug Merck acquired for $1B

Bio Pharma Dive

DECEMBER 15, 2023

Welireg, which Merck got through its purchase of Peloton Therapeutics, can now be used to treat an advanced form of kidney cancer, adding to an earlier clearance in a rare genetic disease.

Genetic Disease

Genetic Disease Genetics Drugs

FDA Refines Accelerated Approval Pathway with New Draft Guidance

XTalks

DECEMBER 9, 2024

The US Food and Drug Administration (FDA) has released a new draft guidance titled Expedited Program for Serious Conditions Accelerated Approval of Drugs and Biologics Guidance for Industry refining the accelerated approval pathway for drugs and biologics targeting serious or life-threatening conditions.

Clinical Trials

Clinical Trials Clinical Trials Compliance Trials

World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing

FDA clears AstraZeneca, Merck drug to treat some genetic breast cancers early

Webinars

Trending Sources

Merck pays Seattle Genetics $1.7B as antibody cancer drugs continue comeback

Webinars

FDA approves Regeneron drug for rare, genetic form of high cholesterol

An AstraZeneca, Merck drug slows the return of genetic breast cancer. Will testing speed up?

Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

FDA clears first-of-its-kind Duchenne drug for broad use

Genetic diagnostics promises paradigm shift towards personalized, proactive healthcare

Ovid turns to gene therapy startup to restock drug pipeline

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Amgen to test new way to lower heart risk with large drug trial

Seattle Genetics stays step ahead of rivals with bladder cancer data

Merck wins approval for cancer drug acquired in 2019 biotech buyout

Indian healthcare looks at pharmacogenomics in patient care as comprehensive genetic tests gain ground

Ionis, AstraZeneca change a key trial, fueling more questions about a genetic heart disease

On the hunt for new ALS drugs, researchers see progress, and a long road ahead

Regeneron, in search of an eye gene therapy, turns to a young biotech

STAT+: Emerging class of genetically targeted drugs induce remissions in leukemia patients

SOPHiA GENETICS and AstraZeneca partner for oncology drugs development

UniQure moves Huntington's gene therapy to next phase of key trial

Synlogic’s SYNB1934 receives orphan drug status from US FDA

Gene therapy startup emerges with green light for first-of-its-kind trial

UK agency pilots biobank to study links between genetics and drug side effects

Lilly adds to genetic meds push with Genetic Leap alliance

Lilly gene therapy finding puts focus on hearing loss treatment pipeline

Biogen shelves ALS drug after early-stage trial failure

Mirum drug for rare genetic disease gains FDA approval

Fulgent Genetics acquires therapeutics development company Fulgent Pharma

“Game-changing” obesity drug over-promising?

UCB enters drug discovery collaboration with Aitia

FDA grants orphan drug designation to DTx Pharma’s CMT1A therapeutic

Indian pharma to take advantage of GenAI potential to streamline drug discovery, reduce cost & time-to-market

Alkeus raises funds to launch Stargardt disease therapy gildeuretinol

Stoke shares surge on updated results for Dravet drug

Leading innovators in genetically modified animal models for the pharmaceutical industry

Mirai Bio launches with plan to help genetic med developers

Integrating Bioanalytical Automation, Biomarkers, Pharmacogenomics, & Cost-Effective Strategies

Pharmacognosy and Drug Patents: Unveiling Nature’s Medicinal Treasures

Gene Therapy and Pharmacokinetics

BridgeBio data show sustained benefit for achondroplasia drug

Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

No drugs for prions: could new approaches alleviate therapeutic scarcity?

FDA widens use of cancer drug Merck acquired for $1B

FDA Refines Accelerated Approval Pathway with New Draft Guidance

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