Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

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Drug Discovery World

APRIL 17, 2024

JMB: There’s no doubt that it’s been a tough environment to raise funds, and the market has only worsened in 2023. DS: You say on your website that your approach is applicable to any disease where increasing protein expression is beneficial. What diseases are you currently targeting? Can you give some examples?

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The Pharma Data

JANUARY 17, 2021

But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion. Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases.

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pharmaphorum

DECEMBER 8, 2021

If approved, the drug will be sold by Neuren Pharma, which licensed North American rights to the drug in 2018 and is also developing another drug for Rett blarcamesine, in collaboration with Anavex Life Sciences. The post Acadia closer to bringing first Rett syndrome drug to market appeared first on.

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Pharmaceutical Technology

JULY 29, 2022

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Drugs for dialysis, amino acid supplements, and drugs which convert blood ammonia are marketed right now as the go-to urea cycle disorder treatments. Future Possibilities.

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Drug Discovery World

JANUARY 30, 2024

“Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Developing more efficient and affordable alternatives is key. Developing value-based payment models that consider long-term benefits could improve access.

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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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XTalks

MAY 17, 2023

The Bespoke Gene Therapy Consortium was jointly launched by the NIH and US Food and Drug Administration (FDA) in 2021 to help address common issues that gene therapy developers face. It included formulating a standardized therapeutic development model that would help accelerate clinical trial processes for new treatments.

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Drug Discovery World

FEBRUARY 15, 2024

Hypothetically, any mRNA-based drug using S-Ac7-DOG as the lipid base would therefore have improved efficacy and a better safety profile.

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pharmaphorum

SEPTEMBER 17, 2020

The UK big pharma hasn’t revealed how much it is paying for rights to the drug candidate, originally developed by US biotech Dogma Therapeutics, which is still in preclinical development. At the moment no oral PCSK9 drugs have started clinical development, and AZ says it intends to start clinical testing of Dogma’s candidate next year.

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pharmaphorum

OCTOBER 4, 2020

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

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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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pharmaphorum

OCTOBER 4, 2022

US-based LogicBio is a clinical-stage genetic medicine company focused on genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. Instead of traditional CRISPR- based gene therapy, the company uses homologous recombination as the basis of its genome editing platform, ‘GeneRide’.

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Delveinsight

JANUARY 12, 2021

Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. AvantGen has announced the licensing of a panel of its anti-SARS-CoV-2 antibody clones to IGM Biosciences for COVID-19 therapy development. R on macrophages.

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Drug Discovery World

AUGUST 28, 2023

PH : This is a relatively new area of biology and it takes many years to get a new drug to the market. 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?

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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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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. It’s much more complex and it has to be 100% correct, or else patients are really at risk,” explains Michael MacNeir, vice president of Business Development, Sharp Clinical.

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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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pharmaphorum

AUGUST 24, 2021

The latest deal allows Vertex to tap into Arbor’s technology platform to develop cell therapies for diseases like type 1 diabetes and blood disorders sickle cell disease (SCD) and beta thalassaemia.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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The Pharma Data

NOVEMBER 19, 2020

He has pioneered the establishment of diagnostic evaluation algorithms for children with sensorineural hearing loss and developed a next generation sequencing platform to determine the genetic causes of hearing loss in children. He has 92 peer review articles published with the majority related to hearing loss. About Sensorion.

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pharmaphorum

JANUARY 9, 2023

Along with Zolgensma – which made its debut in 2019 – Biogen has had approval to market its antisense-based therapy Spinraza (nusinersen) since 2016, while Roche got a green light for its orally-administered therapy Evrysdi (risdiplam) in 2020. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

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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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XTalks

DECEMBER 29, 2022

This highlights potentially new marketing trends that life science companies may have to start looking into if they haven’t already. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. 3. Manufacturing & Supply Chain: Addressing Drug Shortages.

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The Pharma Data

NOVEMBER 29, 2020

The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs. Typically, it is children who suffer from the disease. It was accepted for priority review on June 2. The submission was based on the safety and efficacy data of the pivotal Phase II trials 201 and 12-230.

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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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The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Sigilon Therapeutics

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XTalks

NOVEMBER 10, 2023

Acute manifestations include thrombocytopenia, microangiopathic hemolytic anemia, bleeding episodes, headache and abdominal pain, while chronic ones include stroke, cardiovascular disease and damage to organs. As a rare disease, cTTP affects less than 1,000 individuals in the US.

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pharmaphorum

JUNE 28, 2022

Type A or neuronopathic forms of the disease involve damage to the brain, while type B mostly affects other parts of the body like the lungs, liver and spleen and type A/B is a combination of the two. Children with ASMD type A usually die before their third birthday, while the median life expectancy for type B patients is 17 years.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective?

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The Pharma Data

DECEMBER 9, 2020

Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes.

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XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Rett syndrome is characterized by a distinctive pattern of symptoms that gradually emerge after a period of seemingly normal development. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

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pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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Delveinsight

AUGUST 17, 2021

Food and Drug Administration, which could have entered the ultra-rare genetic disease market. The rarity of disease – 1.36 It is an oral solution for the treatment of Idiopathic Hypersomnia in Adults and it is expected to enter the market later this year for medicinal use. person/million around the globe.

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pharmaphorum

OCTOBER 16, 2020

With large datasets crucial to gaining an understanding of rare diseases and opening the door to drug development, digital technology is proving transformative. Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise. About the a uthors.

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The Pharma Data

DECEMBER 17, 2020

will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. www.lysogene.com.

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The Pharma Data

NOVEMBER 8, 2020

The active ingredient is violaxazine hydrochloride, which has an extensive safety record in Europe, where it was previously marketed as an antidepressant. Reese, executive vice president of Research and Development at Amgen. Celgene acquired it in 2018 from bluebird bio, who originally developed the therapy.

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