pharmaphorum

OCTOBER 27, 2020

Catabasis has conceded defeat with its Duchenne muscular dystrophy drug edasalonexent, pulling the plug on the drug after a phase 3 trial echoed the results of a failed mid-stage study. The post Catabasis craters after giving up on muscular dystrophy lead appeared first on.

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Intouch Solutions

APRIL 12, 2022

Great news for Intouch: The One Show just published their 2022 shortlist of finalists, and we’re well represented with 14 nominations. Even more good news: Intouch work has also made the shortlist with three more nominations for The One Club’s ADC Awards. When they move their heads, the animal moves theirs.

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Camargo

MARCH 9, 2021

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. In the News: March 2021 Regulatory and Development Updates. In the News: February 2021 Regulatory and Development Updates.

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pharmaphorum

JUNE 9, 2021

For RBC’s Brian Abrahams, other potential winners from a more accommodating FDA could be Sarepta – which is developing gene therapies for Duchenne muscular dystrophy – as well as Intra-Cellular Therapies whose Caplyta (lumateperone) is under review at the FDA for bipolar depression.

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Intouch Solutions

FEBRUARY 3, 2022

When a lauded, globally circulated industry magazine known for obsessively curating the best in advertising names your company a 2021 Top Five Creative Agency – and one of the top 15 in the world – you have no choice but to share the news. ” ~ Susan Perlbachs, Chief Creative Officer.

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Delveinsight

AUGUST 27, 2021

Duchenne muscular dystrophy (DMD) is a rare genetic disorder usually diagnosed in young boys, gradually weakening muscles across the body until the heart or lungs fail. What are the currently available treatment options for patients with Duchenne Muscular Dystrophy ( DMD)?

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The Pharma Data

MARCH 22, 2021

This is very unfortunate news to deliver on the tominersen Phase III study and we know it will be especially difficult for people with Huntington’s disease to hear. Dosing will be paused in the open-label extension study (GEN-EXTEND) of tominersen while data are carefully analysed to inform next steps on this study. said Levi Garraway, M.D.,

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pharmaphorum

JULY 11, 2021

It also comes two weeks after STAT News reported “off the books” meetings between Biogen executives and FDA staffers including Office of Neuroscience director Billy Dunn. ” The letter is a change of tone from Woodcock, who initially backed the decision and said it was “a very solid accelerated approval.”

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Delveinsight

JULY 28, 2020

The trial is evaluating SGT-001, which is a novel adeno-associated viral vector-mediated gene transfer indicated for Duchenne muscular dystrophy. Solid Biosciences announced the decision of the US FDA to maintain a clinical hold on Solid’s Phase I/II clinical trial, IGNITE, for DMD.

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XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. The global incidence of DMD is approximately one in every 3,500 to 6,000 male births.

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The Pharma Data

JANUARY 10, 2021

The new year began with a fairly low level of clinical trial news. Sarepta Therapeutics announced topline results from Part 1 of Study SRP-9001-102 for its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD). Here’s a look. COVID-19-Related. Cerecor announced results from its exploratory Phase II U.S.

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The Pharma Data

MAY 17, 2023

I am encouraged by this clinical data for fenebrutinib, which is important news for people living with MS,” said Levi Garraway, M.D., Additionally, pre-clinical data have shown fenebrutinib to be potent and highly selective, and it is the only reversible inhibitor currently in Phase III trials for MS. “I

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STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. Soon after Cure Rare Disease announced that positive news in August, Richard Horgan told the Globe that his brother should “soon” get the treatment.

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XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA).

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pharmaphorum

OCTOBER 27, 2022

Santhera has completed a rolling application for its Duchenne muscular dystrophy (DMD) therapy vamorolone in the US, setting up a possible approval and launch in the latter half of 2023. The post Santhera seeks speedy FDA review of Duchenne drug vamorolone appeared first on.

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pharmaphorum

SEPTEMBER 30, 2022

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the middle of 2023. The post Sarepta files Duchenne muscular dystrophy gene therapy with FDA appeared first on.

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pharmaphorum

OCTOBER 4, 2022

Its key assets include product candidates for Duchenne muscular dystrophy (DMD), Friedreich’s ataxia, and BAG3 mediated dilated cardiomyopathy, among others. AavantiBio, a precision genetic medicine company that was bankrolled by Solid’s rival Sarepta back in 2020, focuses on neuromuscular and cardiac rare diseases.

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pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023.

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pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development.

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pharmaphorum

OCTOBER 3, 2022

In draft guidance published on Friday, it emerged NICE does not recommend PTC Therapeutics’ Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin.

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pharmaphorum

APRIL 19, 2022

Just days after dosing its first patient in a Duchenne muscular dystrophy trial, US biotech PepGen has pushed the button on an initial public offering (IPO). The Boston-based company has filed with the US Securities and Exchange Commission to raise up to $115 million from the IPO, according to a Renaissance Capital report.

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski has advanced therapeutics into human clinical trials for haemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and heart failure. His work remains a key component of every FDA-approved AAV therapeutic and will remain central to future advances.

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pharmaphorum

JANUARY 12, 2022

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. “We will continue to move as quickly as possible to bring SRP-9001 to patients in the US and around the world.”

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pharmaphorum

NOVEMBER 23, 2021

A year ago, Swiss biotech Santhera was slashing staff to cut costs after it abandoned lead Duchenne muscular dystrophy (DMD) therapy idebenone. Now, it’s ready to file replacement drug vamorolone, armed with new data from a pivotal trial, in what looks like a reversal of its fortunes.

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pharmaphorum

JUNE 24, 2022

Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of its new-generation Duchenne muscular dystrophy candidate, after a patient had a serious safety incident. The post Sarepta shares yo-yo as FDA places hold on Duchenne trial appeared first on.

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pharmaphorum

JANUARY 23, 2023

The work on the wearable tech is described in two papers published in the journal Nature Medicine , focusing on Duchenne muscular dystrophy (DMD) and Friedreich’s ataxia (FA) – two rare, degenerative disorders that both lead to disrupted movement patterns, disability, and paralysis.

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pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. There are already several drugs on the market to treat Duchenne, notably from Sarepta Therapeutics which has three FDA-approved drugs that use “exon skipping” technology.

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pharmaphorum

JULY 7, 2022

New data on Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. Roche has exclusive rights to the therapy outside the US.

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pharmaphorum

SEPTEMBER 27, 2021

Capricor Therapeutics’ bid to find a partner for its Duchenne muscular dystrophy (DMD) cell therapy CAP-1002 could get a boost after final phase 2 data showed a strong effect on disease progression. The post Capricor preps phase 3 for Duchenne cell therapy as it seeks a partner appeared first on.

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Scienmag

APRIL 13, 2022

A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles–including the heart muscle–in Duchenne muscular dystrophy patients, a new study published in the international peer-reviewed journal The Lancet shows.

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pharmaphorum

DECEMBER 22, 2021

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. The post Pfizer pauses Duchenne gene therapy trial after patient death appeared first on.

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pharmaphorum

AUGUST 13, 2020

The FDA has approved a new therapy for the rare muscle wasting disease Duchenne muscular dystrophy (DMD) as Japan’s NS Pharma takes on Sarepta and its controversially approved rival.

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XTalks

DECEMBER 24, 2020

There was good news from Chinese vaccines and the Serum Institute of India is on the course of producing the AstraZeneca/Oxford vaccine, so it is not a zero-sum game. In non-pandemic news, it was a big year for CRISPR, the reigning genetic technology in molecular biology since it first debuted in 2012. CRISPR Therapeutics.

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The Pharma Data

JANUARY 3, 2021

It’s not particularly surprising that there wasn’t a lot of clinical trial news between Christmas and New Year’s, although there was some. CAP-1002 is an allogeneic cell therapy in development for Duchenne muscular dystrophy and the cytokine storm associated with COVID-19. Here’s a look. COVID-19-Related.

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