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…

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pharmaphorum

JUNE 9, 2023

3rd Rare & Genetic Kidney Disease Drug Development Summit Mike.Hammerton Fri, 09/06/2023 - 07:13 Bookmark this

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pharmaphorum

JANUARY 24, 2023

Drug development has long been an issue for the pharma industry, due to the expense and the high failure rate of potential treatments. Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates.

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Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). This would allow the companies to boost the development of new therapy options for Rett syndrome and GAN patients.

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Pharma Mirror

APRIL 28, 2021

Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinical trials in the U.S. and Europe. Catalent will further support process optimization and look to reduce material.

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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.

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pharmaphorum

MARCH 21, 2022

Neuroscience is arguably the next great frontier in medicine and 2022 dawned with a wave of exhilarating progress for the neurodegenerative drug development community. A startup led by Novartis veteran raises $80 million to develop treatments for people with degenerative brain conditions.

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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. This will improve accessibility to services for AAV-mediated gene therapy programmes. This will improve accessibility to services for AAV-mediated gene therapy programmes.

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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.

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Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Shilling was appointed to represent the company on the steering committee of the BGTC.

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Pharmaceutical Technology

OCTOBER 19, 2022

Eli Lilly and Company has signed a definitive agreement for the acquisition of all outstanding shares of precision genetic medicine firm Akouos for a total deal value of up to nearly $610m or up to $15.50 Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss.

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Pharmaceutical Technology

FEBRUARY 9, 2023

The collaboration is aimed at discovering and developing new advanced treatments for genitourinary (GU) tumours as well as other related serious diseases using ReviR’s RNA-targeted technologies. ReviR combines computational and high throughput drug discovery technologies to deliver advanced medicines to patients.

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Pharmaceutical Technology

JUNE 10, 2023

CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. According to GlobalData, Phase I drugs for Brain Tumor does not have sufficient historical data to build an indication benchmark PTSR for Phase I. Buy the report here.

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Pharmaceutical Technology

JUNE 10, 2023

CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. According to GlobalData, Phase I drugs for Brain Tumor does not have sufficient historical data to build an indication benchmark PTSR for Phase I. Buy the report here.

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XTalks

MAY 17, 2024

Xtalks spoke with Richard Philipson, MD, Chief Medical Officer (CMO) at Calliditas Therapeutics, a Sweden-based company with full commercial operations in the US focused on developing treatments for rare diseases to learn more about the continuing evolution of rare disease drug development.

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FDA Law Blog

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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Medical Xpress

FEBRUARY 21, 2023

Researchers from Children's Hospital of Philadelphia (CHOP) developed two new zebrafish models for studying a specific genetic form of mitochondrial disease that represents the most common cause of Leigh syndrome. The findings were recently published in the journal Human Molecular Genetics.

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Pharma Mirror

FEBRUARY 23, 2021

Catalent is also announcing the launch of pDNA development and manufacturing services at its Rockville, Maryland facility. The post Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site appeared first on Pharma Mirror Magazine. SOMERSET, N.J. SOMERSET, N.J.

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Pharmaceutical Technology

JUNE 15, 2023

The need for new medical treatments and drugs has never been greater. But before pharmaceutical companies can go to market with a breakthrough drug, they need to ensure safety and efficacy through clinical trials. Even reaching the clinical trial phase offers no guarantees, as only 12% of such drugs receive U.S.

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pharmaphorum

MARCH 26, 2021

There’s a new kid on the block among companies using artificial intelligence in drug discovery, after 1910 Genetics launched today with $26 million in financing. 1910 Genetics is named after the year in which sickle cell disease (SCD) was first discovered in the US by James Herrick. Dr Jen Nwankwo.

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Roots Analysis

JANUARY 19, 2024

Introduction In a strategic move to accelerate the development and launch of genetic medicines, BridgeBio Pharma has successfully secured up to USD 1.25 Acoramidis: A Potential Game-Changer for BridgeBio Pharma in ATTR Treatment Acoramidis, currently under FDA review, represents BridgeBio Pharma’s flagship drug in the ATTR space.

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Eye on FDA

MARCH 7, 2024

Back in December 2023, FDA announced intention in the Federal Register and in a press release to form a new FDA Advisory Committee to be called the Genetic Metabolic Diseases Advisory Committee (GeMDAC). The vote of any committee is advisory in nature, and not binding on the agency’s final decision.

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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.

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XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. The global incidence of DMD is approximately one in every 3,500 to 6,000 male births.

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Drug Discovery World

JANUARY 19, 2024

UK scientists have uncovered the interplay between cancer-driving genetic mutations and inherited genetic variants in a rare type of blood cancer. In the future, this knowledge could aid drug development and interventions that reduce the risk of disease.

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Outsourcing Pharma

JANUARY 23, 2024

Sano Genetics says it is developing software that will enable âthe precision medicine revolutionâ and has raised $11.4 million in new funding.

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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.

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Drug Discovery World

JULY 14, 2023

University of Queensland (UQ) researchers have used genetics to reveal that much of the risk of developing the common and sometimes fatal diverticular disease of intestine (DivD) is inherited. The study has identified new drug targets and opened up possibilities for novel treatment strategies.

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Drug Discovery World

JUNE 8, 2023

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business. In this latest episode, DDW’s Megan Thomas is in conversation with Dr Pierre Eftehkari, Chief Sceitnific Officer of Inoviem Scientific.

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STAT News

DECEMBER 13, 2022

A new study suggests that the root of the problem is buried in our genetic code. Researchers uncovered four gene variants connected with the immune system that significantly increase a person’s risk of developing the disease, known as progressive multifocal leukoencephalopathy or PML. Read the rest…

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pharmaphorum

MARCH 10, 2022

18 months after signing its first partnering deal with Genentech, Scenic Biotech has raised $31 million in first-round financing that it will use to take its own genetic modifier-targeted medicines into clinical trials. Scenic has developed a series of small-molecule drugs targeting QPTCL that block the expression of CD47 on cancer cells.

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pharmaphorum

SEPTEMBER 14, 2020

Merck & Co has signed a multi-billion cancer drug development deal, which will see it investing $1 billion in partner Seattle Genetics. US-based Merck, known as MSD outside North America, will globally develop and market Seattle’s investigational antibody-drug conjugate (ladiratuzumab vedotin).

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BioTech 365

AUGUST 19, 2021

Achromatopsia (Genetic Disorders) Drugs in Development, 2021 Market Research Report – ResearchAndMarkets.com Achromatopsia (Genetic Disorders) Drugs in Development, 2021 Market Research Report – ResearchAndMarkets.com DUBLIN–(BUSINESS WIRE)–The “Achromatopsia (Genetic Disorders) – Drugs in Development, 2021” (..)

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Drug Discovery World

FEBRUARY 28, 2024

Lu Rahman selects some of the year’s interesting and noteworthy advances in cancer research drug discovery and development. As always, a year within cancer drug discovery and development brings with it significant breakthroughs, and 2023 has been no exception. Research into new cancer drugs remain strong.

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Development Research Drugs Antibody 64

Cloudbyz

MARCH 5, 2024

In the intricate dance of drug discovery and development, two protagonists emerge as the cornerstones of modern medicine: biologics and small molecules. Manufacturing Process : Small Molecules: Manufactured through chemical synthesis, small molecule drugs can be consistently reproduced with a high degree of purity and stability.

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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.

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Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics.

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