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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Pharmaceutical Technology

AUGUST 17, 2022

Merck will also make royalty payments on any approved products developed out of the partnership. The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas.

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Pharmaceutical Technology

FEBRUARY 28, 2023

Xinvento will also receive up to $150m in certain commercial net sales milestones related to the lead product or a second molecule in the event of a second molecule’s selection, development and approval.

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Genetic Disease Genetics Sales Development 130

Pharmaceutical Technology

MARCH 1, 2023

Under the agreement terms, Tevard will receive up-front, option-exercise, as well as milestone payments, along with royalties on any products that are approved. The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases.

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Development Gene Therapy Genetics Genetic Disease 130

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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Genetic Disease Genetics Medicine Production 52

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. Life Edit will get an upfront payment and is eligible for potential milestone payments.

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Gene Editing Gene In-Vivo Gene Therapy 147

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

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. You have very little supply and the product is incredibly temperature sensitive,” explains MacNeir. “If

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene Dermatology FDA Approval 97

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

BioTech 365

OCTOBER 28, 2020

NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced the pricing of its underwritten public offering … Continue reading → NOVATO, Calif.,

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BioTech 365

OCTOBER 21, 2020

NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a … Continue reading → NOVATO, Calif. , 21, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc.

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Genetic Disease Genetics Production Development 40

BioTech 365

SEPTEMBER 25, 2020

NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. NOVATO, Calif., 25, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. Kakkis, M.D., Ph.D., … Continue reading →

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Gene Therapy Genetic Disease Gene Genetics 40

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

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Genetic Disease Protein RNA Doctor 104

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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Drugs Gene Therapy Genetic Disease Gene 98

pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule.

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RNA Genetics Dermatology Antibody 105

Pharmaceutical Technology

APRIL 4, 2023

Orphacol® is a pharmaceutical product containing cholic acid, a substance found in the bile, which is used to digest fats. Orphacol® is used to treat adults and children who suffer from Bile Acid Synthesis Disorders (BASD), a genetic abnormality that makes them unable to produce bile. For more information on CTRS, please go to [link].

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XTalks

MAY 17, 2023

Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall “The BGTC offers promise for rare disease patients around the world,” said Julie Gerberding, MD, CEO of the FNIH, in the press release from the agency. In the US, more than 30 million people live with a rare disease.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

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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Gene Therapy Gene Genetic Disease Manufacturing 66

The Pharma Data

OCTOBER 27, 2020

. “Agena proactively sourced and secured materials to provide uninterrupted product availability to our customers. Agena Bioscience develops, manufactures, and supplies genetic analysis systems and reagents, including the MassARRAY ® System. ” More information is available at www.agenabio.com. About Agena Bioscience.

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Genetic Analysis Genome Genomics Reagent 52

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. The body relies on this enzyme to break down glycogen, a stored form of sugar utilized for energy production.

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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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Pharmaceutical Technology

APRIL 24, 2023

Due to their size, cells can absorb them where they release products. He also says this is great for drugs like Bristol Myers Squibb’s Opdivo (nivolumab) and Merck’s Keytruda (pembrolizumab), which target cell surface receptors, but it is difficult to select a range of other targets to treat diseases.

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Drug Delivery Vaccine Vaccination Research 147

pharmaphorum

AUGUST 1, 2022

There has been speculation that muscle weakness and/or myocarditis may be a class risk for gene therapies that are delivered using adeno-associated virus (AAV) vectors, and work is ongoing to see if the risk could be mitigated by identifying genetic risk factors or preventative treatments.

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Gene Therapy Gene Genetics Trials 105

pharmaphorum

OCTOBER 16, 2020

These techniques make the production of and access to high-quality data on rare diseases the gateway to treatment identification, and so are proving more crucial than ever for organisations in pharma. Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise.

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Trials Clinical Trials Clinical Trials Life Science 105

The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

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Protein RNA Production Genetics 52

pharmaphorum

DECEMBER 6, 2020

The trials are the first to test a CRISPR/Cas9 gene editing therapy in humans for a genetic disease, according to the partners. By gene editing the patient’s own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases,” he added.

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Gene Editing Gene In-Vivo Genetics 52

Roots Analysis

FEBRUARY 21, 2022

However, the conventional nucleotide library preparation process has several challenges, including requirement of huge amount of starting materials, inadequate throughput and deteriorated quality of the product. Product Competitiveness Analysis. Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis.

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Marketing Gene Sequencing DNA Genomics 52

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases. Any products from the collaboration will be developed and commercialized by Roche. Rare disease data-focused AllStripes raises USD 50 Million to enhance medical record mining.

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Vaccine Vaccination Gene Therapy RNA 98

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug.”.

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FDA Approval Protein Containment Genetic Disease 64

The Pharma Data

NOVEMBER 29, 2020

PH1 is an ultra-rare genetic disease marked by overproduction of oxalate, which causes deposits of calcium oxalate crystals in the kidneys and urinary tract and can lead to painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and system organ dysfunction. Oxlumo decreases oxalate production.

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Genetic Disease Antibody Licensing Licensing 52

The Pharma Data

DECEMBER 3, 2020

In the Phase 3 trial, the product was safe and well tolerated. In October, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) granted ORLADEYO a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). The most frequently reported adverse reactions were gastrointestinal related.

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FDA Approval Medicine Genetic Disease Trials 52

The Pharma Data

JANUARY 26, 2021

The advent of biologically active agents has paved way to new treatment avenues in the developmental and genetic disease, thereby boosting the orthopedics market. Rising burden of orthopedic diseases in developed countries has spurred new research in the global orthopedics market. Orthopedics Market: Regional Assessment.

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Marketing Development Genetic Disease Research 52

XTalks

JANUARY 3, 2025

FCS is a rare genetic disorder that prevents the body from properly breaking down triglycerides (a type of fat in the blood), leading to dangerously high levels. By lowering its production, Tryngolza significantly reduces triglyceride levels in the blood.

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FDA Approval Drugs Clinical Trials Clinical Trials 59

The Pharma Data

DECEMBER 9, 2020

NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00

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Gene Therapy Genetics Genetic Disease Sales 40

XTalks

SEPTEMBER 17, 2020

In the case of the former, pharmaceutical and biologics companies would be eligible for approval of their product through traditional regulatory pathways; the latter could support accelerated approval. Symptoms and Etiology: Cushing’s disease is a disorder caused by excess cortisol which is often the result of a pituitary adenoma.

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Trials Gene Hormones Clinical Trials 98

pharmaphorum

APRIL 20, 2022

Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Why cancer?

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Gene Editing DNA Gene Genetics 52