Pharmaceutical Technology

JANUARY 10, 2023

The multi-year collaboration will focus on discovering precision genetic medicines by developing a new platform for complete gene insertion by delivering a single vector of gene editing and DNA cargo. Beyond the liver, ReCode’s SORT LNP genetic medicines technology enables the delivery to target cells and organs.

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XTalks

SEPTEMBER 27, 2024

In a news release published yesterday, Pfizer announced it will voluntarily withdraw its sickle cell disease (SCD) therapy Oxbryta (voxelotor) from all markets worldwide. Pfizer said it has notified regulatory authorities of the market withdrawal. This is a significant turn of events as Pfizer acquired the drug in the $5.4

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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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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). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.

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BioPharma Reporter

JULY 21, 2022

US biotech, CAMP4 Therapeutics, has secured US$100m in a Series B financing round that it says will be used to accelerate expansion of its regulatory RNA (regRNA) platform.

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Pharmaceutical Technology

MARCH 1, 2023

The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases. It is exploring the use of its new Enhancer tRNA, mRNA Amplifier, and Suppressor tRNA platforms in heart disease, muscular dystrophies, and neurological disorders.

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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. In addition to its clinical promise, Mirums FDA approval of Ctexli has significantly bolstered its market position.

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Outsourcing Pharma

OCTOBER 22, 2024

The European Medicines Agencyâs Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna (ataluren), PTC Therapeuticsâ treatment for the genetic disease Duchenne muscular dystrophy (DMD).

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Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases.

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BioTech 365

OCTOBER 5, 2020

Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight, leading to the Marketing Authorisation Application for LUMEVOQ LUMEVOQ is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary … Continue reading →

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The Pharma Data

NOVEMBER 19, 2020

MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. Dr. John Greinwald is a tenured professor of Otolaryngology and Pediatrics with over 20 years of experience with a focus on the genetic causes and treatment of deafness. To register for the call, please click here.

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Genetics Containment Genetic Disease Gene 52

The Pharma Data

JANUARY 26, 2021

Orthopedics Market: Overview. Strides in the orthopedics market have been primarily driven by changing demographic profile of patient populations and advances made in orthopedic surgeries. Concomitant research in biomaterials, biomechanics, electrophysiology, and molecular biology has expanded the horizon of the orthopedics market.

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pharmaphorum

JANUARY 9, 2023

First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.

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XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). This includes a greater focus on digital campaigns and diversifying marketing campaigns with race and ethnicity insights.

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Life Science Genetic Disease FDA Approval Marketing 98

pharmaphorum

NOVEMBER 27, 2020

CF is a rare, life-shortening genetic disease that affects the lungs, liver, gastrointestinal tract, sinuses, sweat glands, pancreas and reproductive tract. It is caused by a defective or missing CFTR gene, and there are around 75,000 people worldwide with the disease.

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pharmaphorum

AUGUST 12, 2020

The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8

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Drugs Genetic Disease Gene Therapy Trials 98

Roots Analysis

FEBRUARY 21, 2022

The various advantages of NGS are briefly described below: Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis. Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis. Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis.

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Marketing Gene Sequencing DNA Genomics 52

Pharmaceutical Technology

APRIL 4, 2023

Abacus Medicine Pharma Services (AMPS) and Laboratoires CTRS, marketing authorization holder of Orphacol®, are proud to announce a strategic partnership that ensures continuous stock of Orphacol® in Spain. Orphacol® is a pharmaceutical product containing cholic acid, a substance found in the bile, which is used to digest fats.

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XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes.

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Clinical Trials Clinical Trials Genetics Trials 98

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company.

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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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XTalks

MAY 17, 2023

In the past several years, gene therapies have finally been hitting the market after decades of setbacks and uncertainties. In 2022, the FDA approved four new gene therapies, and more than a handful are looking to get approval this year for diseases including Duchenne muscular dystrophy (DMD), sickle cell disease (SCD) and hemophilia A.

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pharmaphorum

FEBRUARY 17, 2022

The drug – which costs $125,000 at US list prices – can be used either alone or in combination with hydroxyurea, a well-established therapy for the disease. The confounder for both GBT and Novartis’ hopes for their drug could be genetic therapies for SCD, which offer a one-shot treatment for the disease.

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Roots Analysis

AUGUST 12, 2024

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

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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. As per the agreement, Alexion will initiate a cash tender offer through a subsidiary to acquire all outstanding shares of LogicBio for $2.07

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pharmaphorum

DECEMBER 8, 2021

The post Acadia closer to bringing first Rett syndrome drug to market appeared first on. Another late-stage candidate – Newron Pharma’s sarizotan – failed a late-stage trial and was axed by its developer last year.

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pharmaphorum

AUGUST 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans.

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Gene Therapy Gene Genetics Trials 105

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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Gene Editing Gene In-Vivo Clinical Trials 59

pharmaphorum

SEPTEMBER 17, 2020

There are two antibodies already on the market that target PCSK9 – Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but these require subcutaneous injection every two to four weeks.

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The Pharma Data

OCTOBER 27, 2020

“The MassARRAY SARS-CoV-2 Panel is the only assay on the market with 5 viral genome targets multiplexed into a single reaction, making it robust against potential mutations arising in the viral genome,” said Dr. Darryl Irwin , Vice President of Scientific Affairs.

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XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene. Rett syndrome is a neurodevelopmental disorder — not a neurodegenerative disease.

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pharmaphorum

AUGUST 24, 2021

According to the terms of the agreement, Arbor is in line for an undisclosed upfront payment and equity investment, plus milestone payments tied to a maximum of seven programmes and royalties on sales if any reach the market.

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Gene Editing Gene In-Vivo Genetics 52

pharmaphorum

JANUARY 11, 2021

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly.

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XTalks

NOVEMBER 27, 2023

Pompe disease, also referred to as acid-maltase disease and glycogen storage disease II, is an uncommon genetic disorder characterized by the gradual weakening of both cardiac and skeletal muscles. Crowley, executive chairman of Amicus Therapeutics, in the company’s press release.

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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years.

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pharmaphorum

DECEMBER 9, 2021

An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. ” Trading in Reata’s shares was halted ahead of the meeting, but the stock was down 38% pre-market this morning. .”

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pharmaphorum

OCTOBER 16, 2020

Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise. Over the last five years we have focused on the power of drug repurposing for rare genetic diseases. The post Rare diseases, repurposing and the role of AI appeared first on. About the a uthors.

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