Drug Discovery World

JUNE 22, 2023

This is the latest episode of the free DDW narrated podcast, “Challenges and tactics for transformative drug development” It covers two articles written for Volume 23, Issue 2 – Spring 2022 of DDW. They are called “ Balancing speed and quality in CLD for faster drug development ”, and “ Sitting down with BridgeBio Pharma ”.

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Pharmaceutical Technology

MAY 4, 2023

The medicine is currently available for use in more than 30 countries. CF is a rare, life-shortening genetic disease that is estimated to impact over 88,000 people across the globe. The progressive multi-organ disease affects the patients’ lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract.

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Scienmag

FEBRUARY 17, 2021

Credit: Mark Bauer/FSU College of Medicine Nearly a half-million people a year die from sudden cardiac death (SCD) in the U.S. A leading cause of SCD in young athletes is arrhythmogenic cardiomyopathy (ACM), a genetic disease in which healthy heart muscle is replaced over time […].

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Drug Discovery World

JUNE 22, 2023

This is the latest episode of the free DDW narrated podcast, “Challenges and tactics for transformative drug development” It covers two articles written for Volume 23, Issue 2 – Spring 2022 of DDW. They are called “ Balancing speed and quality in CLD for faster drug development ”, and “ Sitting down with BridgeBio Pharma ”.

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Development Drugs Genetic Disease Genetics 52

Scienmag

MAY 2, 2022

WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

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Scienmag

JULY 1, 2022

Curing debilitating genetic diseases is one of the great challenges of modern medicine. During the past decade, development of CRISPR technologies and advancements in genetics research brought new hope for patients and their families, although the safety of these new methods is still of significant concern.

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Drug Discovery World

NOVEMBER 7, 2022

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. DDW’s Megan Thomas observes developments of accessibility in this sector and the potential impact. . The origins .

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Scienmag

JUNE 16, 2021

million grant from The National Institutes of Health to develop methods that will improve genetic risk estimates – polygenic risk scores – for specific diseases in people from diverse populations and mixed ancestries. LOS ANGELES (June 16, 2021) – UCLA Health will receive a $4.8

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Pharmaceutical Technology

MAY 22, 2023

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

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STAT News

FEBRUARY 3, 2023

The Boston drugmaker’s medicines for the genetic disease — which damages the lungs and other organs — have transformed the lives of patients, helping them abandon other time-consuming treatments, shrug off what would have been dangerous respiratory infections, and just live breathing easier.

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Genetic Disease Genetics Medicine Drugs 98

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

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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. Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. “They called him ‘The Boy.’

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Drug Discovery World

DECEMBER 19, 2023

Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).

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Drug Discovery World

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . Understanding rare diseases remains a significant challenge.

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Scienmag

MAY 26, 2022

— CRISPR has ushered in the era of genomic medicine. A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases. ITHACA, N.Y.

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Drug Discovery World

JANUARY 24, 2023

DMD and FA are rare, degenerative, genetic diseases that affect movement and eventually lead to paralysis. The researchers estimate that using these disease markers mean that significantly fewer patients are required to develop a new drug when compared to current methods.

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Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.

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Delveinsight

AUGUST 17, 2021

Food and Drug Administration gave a red flag to FibroGen’s anemia pill roxadustat, for the use in patients suffering from kidney diseases owing to safety concerns that arose after results, which in turn are concluded for the presentation of further clinical studies to assess safety for future medicinal use.

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Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).

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World of DTC Marketing

JANUARY 24, 2022

Venture capitalists have poured $42 billion into drug development over the past three years. Most small biotech companies rely on venture capitalist funding to develop new drugs but is that a good way to go? billion, which was much lower than previous studies, which have placed the average cost of drug development as $2.8

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Scienmag

APRIL 6, 2021

Researchers developed a new animal model to study a rare genetic disease that can lead to blindness at the age of 40-50.

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Roots Analysis

FEBRUARY 26, 2024

Literary and archaeological evidences suggest that diseases have plagued living organisms since the beginning of time. Whether it was a common cold or a genetic disease, prayers and sacrifices to the gods were the only hope of a remedy at such times. The science of vaccinology has evolved significantly since Jenner’s discovery.

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The Pharma Data

OCTOBER 21, 2020

NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the company will host a conference call and live webcast on Thursday, November 5, 2020 at 8:00 a.m. CAMBRIDGE, Mass., 22, 2020 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc.

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pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. per share, a rare 660% premium on LogicBio’s share price. .

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XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.

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Drug Discovery World

JULY 11, 2022

However, the speed at which CGT products can impact healthcare depends on how the industry will navigate novel challenges and risks from the nascent field of CGT development. There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. Conclusion. The promise of gene therapy is substantial.

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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. In the US, more than 30 million people live with a rare disease.

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Scienmag

JUNE 23, 2021

A Canadian research team has uncovered a new mechanism involved in Bourneville tuberous sclerosis (BTS), a genetic disease of childhood. The team hypothesizes that a mutation in the TSC1 gene causes neurodevelopmental disorders that develop in conjunction with the disease.

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pharmaphorum

JULY 20, 2022

CAMP4 reckons antisense oligonucleotide (ASO) therapeutics targeting these regRNA sequences have the potential to dial up the production of proteins from any disease-associated gene, treating genetic diseases “at their core.”

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RNA Genetic Disease Drugs Protein 52

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., as Head of Business Development. .–( BUSINESS WIRE )– EdiGene, Inc. as Head of the US Subsidiary, and Kehua Fan, M.D.,

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Drug Discovery World

NOVEMBER 7, 2022

In 2018, the UK’s department of health announced an NHS Genomic Medicine Service, which allows patients with rare diseases to have their entire genetic code read in the hope of providing a much-needed diagnosis. Consequently, the UK has established itself at the forefront of diagnostic whole genome sequencing.

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Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. The requirements When handling personalised medicines, time is of the essence.

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Pharmaceutical Technology

MAY 8, 2023

ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models. According to GlobalData, there are 60+ companies, spanning technology vendors, established pharmaceutical companies, and up-and-coming start-ups engaged in the development and application of zinc-finger nucleases.

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The Pharma Data

AUGUST 9, 2021

“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research.

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The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

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Drug Discovery World

AUGUST 28, 2023

We have a combination of talented and motivated young scientists along with experienced industry experts who have been through the process of drug research and development. Could it be possible to prevent genetic diseases? DS: What’s next for the company’s pipeline? LinkedIn profile.

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The Pharma Data

OCTOBER 27, 2020

The company plans to tackle the existing challenges of precision medicine with a three-prong approach – developing better medicines for known cancer-driving mutations, developing drugs for targets previously thought to be undruggable, and finding new, untapped targets that could lead to even better drugs. Sirnaomics.

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