pharmaphorum

JANUARY 24, 2022

Health Canada has given the drug a green light – under the Sohonos brand – to treat fibrodysplasia ossificans progressiva (FOP), making it the first treatment for the ultra-rare genetic disorder. agonist for both chronic use and flare-ups in FOP, a genetic disorder in which tendons and ligaments gradually become more bone-like.

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XTalks

APRIL 5, 2024

In Europe, the drug, branded Albrioza, was turned down by the European Medicines Agency (EMA), a decision that Amylyx appealed and lost in October 2023. Biogen and Ionis Pharmaceuticals’ antisense drug Qalsody received FDA accelerated approval in April 2023 , but only for a small subtype of ALS patients with a mutation in the SOD1 gene.

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The Pharma Data

MAY 31, 2022

Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality.

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XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. Viral Vectors for Gene Delivery to the CNS. Reporter genes to understand dose and distribution. Nusinersen: A Landmark Therapy.

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XTalks

NOVEMBER 1, 2024

It is a biologic drug available exclusively as a brand-name medication, with no generic or biosimilar forms available. Botox is one of the most prescribed branded treatments for chronic migraine prevention, offering rapid relief with just four treatments per year. Price of Zolgensma: Zolgensma has a price of $2.1

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The Pharma Data

NOVEMBER 8, 2021

Amgen today announced new data from the HER-MES study, the first and only head-to-head study of Aimovig® (erenumab-aooe), a calcitonin gene-related peptide (CGRP) inhibitor, against topiramate for adult patients with episodic and chronic migraine. MBA, trial investigator and managing medical director at Charité Universitätsmedizin in Berlin.

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The Pharma Data

APRIL 29, 2022

It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. About SMA SMA is a severe, progressive neuromuscular disease that can be fatal.

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Protein In-Vitro Medicine Development 52

The Pharma Data

DECEMBER 17, 2020

Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. Prior to SpringWorks, Smith was the executive vice president of Gene Therapy at Bamboo Therapeutics.

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Development Gene Therapy Engineer Life Science 52

The Pharma Data

NOVEMBER 4, 2020

With years of extensive experience in the field of skin health, the Company has successively built the “Juyou” brand for skin barrier repair, now recommended by thousands of public hospitals in China ; the professional baby skincare brand “Juyou Baby”, as well as the “Dermacid” brand, just to name a few.

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Licensing Licensing Dermatology Production 40

XTalks

SEPTEMBER 12, 2023

By fostering a sense of community, addressing pain points and sharing impactful stories, podcasts can strengthen ties with an audience, drive brand loyalty and help life science organizations position themselves for long-term success in an increasingly competitive landscape. Have questions?

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World of DTC Marketing

JANUARY 31, 2021

So where to start… Congresswoman Porter may want to look at the percentage of generic drugs available virus branded (89%). Ex vivo (where cells are genetically modified outside the body) cell and gene therapies have generated considerable excitement on their potential to cure previously incurable diseases.

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pharmaphorum

SEPTEMBER 21, 2021

In other cases, rare CNS conditions may be passed on from one generation to the next without knowledge or understanding that the reason for suffering or death among previous generations was genetic in basis and therefore identifiable, and perhaps even treatable. This is and should be a priority for our health system. About the author.

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Gene Therapy Gene Clinical Trials Clinical Trials 90

The Pharma Data

NOVEMBER 22, 2020

The FDA approved it under the brand name Gavreto on September 4. Both POMC deficiency obesity and LEPR deficiency obesity are ultra-rare genetic disorders. LEPR deficiency obesity is caused by variants in the LEPR gene that often lead to severe obesity starting early in life with insatiable hunger and endocrine abnormalities.

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Licensing Licensing Drugs Gene 52

The Pharma Data

JUNE 24, 2021

“The prognosis for men with advanced prostate cancer has significantly improved since the introduction of novel hormone therapies, but additional therapeutic options are needed for the approximately 25 percent of men with tumors harboring DNA damage response (DDR) gene mutations, who may have poorer outcomes,” said Chris Boshoff, M.D.,

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DNA Clinical Trials Clinical Trials Trials 52

XTalks

FEBRUARY 5, 2024

Alecensa is approved in Japan for the additional indication of recurrent or refractory ALK fusion gene-positive anaplastic large-cell lymphoma. One of the prime reasons for the sales of Zoladex is that it is only available as brand-name medication, and no generic form is available. Alecensa is not currently available in generic form.

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FDA Approval Drugs Sales Development 101

XTalks

APRIL 11, 2024

Similarly, Abecma’s boxed warning includes a description that outlines occurrences of T cell malignancies observed after treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies like Abecma.

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FDA Approval Manufacturing Trials Clinical Trials 52

pharmaphorum

OCTOBER 27, 2022

Alnylam’s earlier plan was to start trials of the drug before the end of this year in the genetic eye disease, which causes fatty material to build up in the macula area of the retina and affect vision. There is currently no approved treatment, although both drug- and gene-based therapies are in development.

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Trials Drugs Clinical Trials Clinical Trials 52

The Pharma Data

DECEMBER 8, 2020

The majority of this revenue growth is expected to come from the company’s Wave 1 pipeline, which includes 12 unique New Molecular Entities (NMEs), representing potential best-in-class/first-in-class therapies, and its existing 14 global brands. Food and Drug Administration (FDA). ABOUT TAK-721 and TAK-003.

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Containment Engineer FDA Approval Vaccination 52

XTalks

FEBRUARY 5, 2024

Alecensa is approved in Japan for the additional indication of recurrent or refractory ALK fusion gene-positive anaplastic large-cell lymphoma. One of the prime reasons for the sales of Zoladex is that it is only available as brand-name medication, and no generic form is available. Alecensa is not currently available in generic form.

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FDA Approval Drugs Sales Development 52

XTalks

FEBRUARY 21, 2025

Encoded Therapeutics Genetic medicines biotech Encoded Therapeutics is reducing its workforce by roughly 29% or about 60 employees from its approximately 200-person team. It said this would lead to a , more efficient company while investing behind growth brands and promising areas of science.

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XTalks

DECEMBER 20, 2023

The companies include a diverse range engaged in innovative approaches in areas like oncology, genetic medicines, inflammatory diseases, dermatology and cardiorenal therapy. report indicating that they accounted for half of the most viewed new brands. Notably, pharmaceutical drugs dominated TV ad impressions this year, with an iSpot.tv

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The Pharma Data

DECEMBER 20, 2020

FLT180a is an AAV gene therapy. Sio Gene Therapies reported positive six-month follow-up data from the low-dose cohort of its dose escalation Phase I/II trial of AXO-AAV-GM1. The product is an AAV9-based gene therapy for GM1 gangliosidosis. The vaccine only requires a single dose. Non-COVID-19-Related.

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Trials Gene Therapy Vaccine Vaccination 52

The Pharma Data

DECEMBER 30, 2020

Another Biden plan opens up foreign markets for consumers to buy their prescription drugs and calls for limiting price increases on branded drugs and generics for drugs included in the Medicare program. It is a strong potential player in the gene therapy space. billion, Regenxbio is also a gene therapy company.

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Vaccine Vaccination Gene Therapy Trials 52

The Pharma Data

MAY 13, 2021

DISCLAIMER: Cory Kelby is a pen name and brand name of letsquitsugar.com. Johnson RK, Appel LJ, Brands M, Howard BV, Lefevre M, Lustig RH, Sacks F, Steffen LM, Wylie-Rosett J; American Heart Association Nutrition Committee of the Council on Nutrition, Physical Activity, and Metabolism and the Council on Epidemiology and Prevention.

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XTalks

SEPTEMBER 14, 2020

CanSino Biologics is developing a vaccine that involves snipping a bit of the virus’ genetic code off and coupling it with a harmless virus to provoke a SARS-CoV-2 immune response. Several laboratories have already shown that they can detect genetic material from the virus in wastewater.

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