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

XTalks

MARCH 19, 2025

This drop likely reflects market concerns over the therapys safety profile. However, since the news broke, Sareptas shares fell about 26.3%, closing at $74.55 on March 18, 2025, where more than 8.2 million shares had exchanged hands in the first few hours of trading according to Reuters.

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Gene Therapy Gene Genetics Clinical Trials 80

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences president and CEO Bo Cumbo said: “This collaboration with Phlox Therapeutics is an exciting opportunity to work with a company that shares our commitment and innovative approach to bringing transformative therapies to patients with rare genetic diseases.

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RNA Gene Therapy Genetics Genetic Disease 130

XTalks

MAY 17, 2023

In the past several years, gene therapies have finally been hitting the market after decades of setbacks and uncertainties. The announcement of its clinical trial portfolio is an important milestone, bringing people with rare genetic diseases one step closer to approved therapies.”

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Gene Therapy Clinical Trials Clinical Trials Gene 98

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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Marketing Drugs Life Science Trials 59

XTalks

FEBRUARY 25, 2025

XTALKS WEBINAR: Key Strategies for Successful Orphan Drug Launch in Europe Live and On-Demand: Friday, March 28, 2025, at 1pm EDT (5pm GMT) Register for this free webinar to learn how to optimize pricing and reimbursement strategies and leverage patient advocacy for a successful market launch of orphan drugs.

FDA Approval 59

FDA Approval Gene Therapy Gene Genetics 59

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.

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Trials Clinical Trials Clinical Trials Life Science 105

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

XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

NOVEMBER 27, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio Price of Pombiliti and Opfolda Therapy Amicus Therapeutics is set to introduce Pombiliti and Opfolda to the US market without delay, with an annual price of $650,000 for patients with a median weight.

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FDA Approval Clinical Trials Clinical Trials Trials 104

The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. Cornering the untapped Chinese CNS market, SciNeuro launched with $100 million in their pocket.

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RNA Antibody Life Science Protein 52

XTalks

MAY 12, 2023

What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.

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FDA Approval Genetics Trials Drugs 97

The Pharma Data

OCTOBER 15, 2020

marketing and diagnostics. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

XTalks

SEPTEMBER 17, 2020

Thiola (tiopronin) is produced by Retrophin, the rare disease biopharmaceutical company founded by Martin Shkreli. The marketing rights to Thiola were acquired by Retrophin in 2014 from Mission Pharmacol.

Trials 98

Trials Gene Hormones Clinical Trials 98

XTalks

SEPTEMBER 16, 2024

Along with the NDA acceptance, KalVista recently announced that the European Medicines Agency (EMA) validated the company’s Marketing Authorization Application (MAA) for sebetralstat. KalVista said it expects to file for approval in the UK, Japan and other countries later in 2024.

Protein 52

Protein Genetics Trials Allergies 52

XTalks

NOVEMBER 10, 2023

Approvals for rare disease treatments have been on the rise in a growing rare disease market that is currently valued at more than $195 billion in 2022. The company received an FDA nod for its VEGFR inhibitor Fruzaqla (fruquitinib) as a third-line treatment for adults with metastatic colorectal cancer.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

SEPTEMBER 24, 2024

Expanding the Treatment Landscape Looking ahead, Marinus aims to broaden its impact on the rare epilepsy market. This patient-centric approach exemplifies Marinus’ commitment to developing treatments that truly meet the needs of those they serve.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Development 52

XTalks

DECEMBER 13, 2023

Moreover, it has submitted global marketing authorization applications for mRNA-1345, a vaccine designed to prevent RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD), supported by favorable data from a predefined interim analysis. vaccine, mRNA-1273.815.

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Genetics Vaccine Vaccination DNA 111

XTalks

FEBRUARY 24, 2025

Despite limited markets for the rare disease drugs, analysts predict that Wainua could reach $700 million in peak sales in just the ATTRv-PN indication. In 2024, Qalsody brought in $32 million, and Wainua generated $85 million in global sales , with $4 million and $20 million in royalties for Ionis, respectively.

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Trials RNA Allergies Drugs 59

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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In-Vivo Marketing Engineer Genetics 52

XTalks

FEBRUARY 27, 2023

Related: Interview with the Leaders at Amylyx, Maker of New FDA Approved ALS Drug Relyvrio – Xtalks Life Science Podcast Ep. 90 Accessing Alpha-Mannosidosis Treatment The Chiesi Group previously received marketing authorization from the European Commission for Lamzede in 2018.

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

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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Gene Therapy Gene Trials Clinical Trials 95

XTalks

JANUARY 6, 2025

Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future. The global market for cell and gene therapies , valued at $5.9

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Gene Therapy RNA Gene Editing Gene 111

XTalks

NOVEMBER 29, 2024

This decision, communicated through a Complete Response Letter, means the drug cannot currently proceed to market. Govorestat is a central nervous system (CNS)-penetrant aldose reductase inhibitor designed to target a critical problem in classic galactosemia — a rare genetic condition that prevents proper metabolism of the sugar galactose.

Drugs 98

Drugs Compliance Clinical Trials Clinical Trials 98

XTalks

FEBRUARY 26, 2025

The CHORD trial, a Phase I/II first-in-human study, involved 12 children aged 10 months to 16 years. Nine participants received an intracochlear injection in one ear, while three received injections in both ears via a procedure similar to cochlear implantation, making it suitable even for young infants.

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Gene Therapy Gene Clinical Trials Clinical Trials 87