Category Archives: Genetic Therapy

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positiveresults and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a program update for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but needed additional dialogue with the agency to confirm.

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FDA knocks back Sarepta in Duchenne gene therapy race with Pfizer, but analysts urge caution - Endpoints News

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Read next: How very tiny technologies are helping tackle the global pandemic

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Your online branding is key to your business success. Heres your roadmap - The Next Web

The owners of a Turkish takeaway in Dunstable are appealing to their customers to help raise 1.9 million for life-altering treatment for their baby cousin, who was diagnosed with a a severe muscle-weakening disease

Metehan Fidan was born in November, and just two months later was diagnosed with spinal muscular atrophy (SMA) type 1, a neuromuscular disease that leads to the muscles weakening over time.

His parents, Tuncay and Zeliha Fidan, who live in Leicestershire, are hoping to raise funds towards a life-altering gene therapy called Zolgensma.

But at $2.1 million (1.9m), Zolgensma is currently valued as the most expensive drug in the world, and is not yet approved in the UK.

The family have started a crowdfunding campaign to raise 1.9 million towards receiving the treatment outside of the UK.

This week, brothers Orhan and Zak Gurbuz - owners of Irmak BBQ in Dunstable - are donating three days revenue from the business to the fundraising page.

To support their baby cousin's treatment, Irmak BBQ will be forwarding all proceeds from sales from Monday, September 14, to Wednesday, September 16.

In a post on facebook, they said: "Irmak BBQ is reaching out to our friends, family, business associates & loyal customers. Our baby cousin needs your help.

"Without everyones help little Metehan might not see his 2nd birthday. We have started a crowdfunding campaign for Metehan Fidan."

On the Go Fund Me page, it says: "We are starting a crowdfunding campaign for Metehan Fidan (born on 12th Nov 2019 at Leicester Royal Infirmary, Leicester, UK) who is the son of my friends Tuncay and Zeliha Fidan both very kind and have always found ways to help their neighbours and the community they live in.

"Unfortunately, Metehan was diagnosed with spinal muscular atrophy (SMA) type 1 a neuromuscular disease so severe that many children with SMA type 1 do not reach the age of two.

"However, there is hope: In order to have a relatively healthy future, Metehan must receive a one-time dose of a gene therapy called Zolgensma.

"As SMA is a rare disease, Novartis charges a huge fee to cover their R&D costs and make a profit: Zolgensma costs $2.1 million with additional expenses such as travel, accommodation and consultancy fees, the total amount of reaches ~$2.45 million (currently ~1.9m).

"For Novartis to approve Zolgensma therapy, a child must not reach 2 years or 13kg as this negatively affects the effectiveness and the safety of the treatment. Metehan is currently 11 months old (as of September 2020), thus we have a fight against the clock.

"The family do not have the means to make up anywhere near the required amount in this time frame, thus they need our help.

"Please donate as much as you can even if its a fiver and give Metehan and his family the chance to give life a shot. Please also share with anyone you feel would be willing to help."

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Dunstable business appeals for help to raise 1.9m for baby cousin with rare genetic disorder - Dunstable Today

This time last year little Hallie Campbell from Wigan was unable to walk, talk or even sit up by herself. She was also suffering daily painful seizures which often went on for hours at a time.She'd been born with an incredibly rare genetic brain condition called AADC deficiency.A bit like Parkinson's disease, it left her effectively locked in her own body.

The first time I met Hallie back in August last year, her family was desperately trying to raise 70,000 for a brand new treatment being offered in Poland.It was so new, it was actually only still being trialled but Hallie's family felt it was their only hope of transforming her life.

I remember that first meeting with the family very well.Despite how desperately ill Hallie was, she still had the most amazing smile.But within minutes of us arriving and beginning to film, she suffered one of her daily seizures and I saw first-hand the distressing reality that she had been living with for the last 6 years.

Her story had such a response when we aired it on Granada Reports that hundreds of our viewers, complete strangers, felt moved to donate to the family's fundraising campaign. In just a few short weeks, they had reached their target and we followed them to Warsaw in Poland where she underwent pioneering gene therapy surgery.We were given exclusive access to film the whole procedure, as Hallie became only the 8th child to have the operation in Poland.

It was risky surgery - brain surgery to be precise - where neurosurgeons drilled into her skull and replaced a faulty gene in Hallie's brain to encourage her body to start producing an enzyme she's deficient in.The operation appeared to go well, but the treatment was so new, her prognosis was uncertain, and no-one knew how effective it would be.

Well fast-forward 10 months and I went back to visit Hallie and her family, to see just how she was doing.And as I walked into their back garden, I couldn't quite believe my eyes.This little girl, who when I last saw her, was in a wheelchair, locked in her own body, was there on a garden chair, sitting up all on her own, smiling that huge smile and waving at me.

The transformation was quite extraordinary.

And that wasn't it.As she grabbed hold of one of her books, she actually spoke."A book" she said.And just like a baby begins to reach each new milestone, Hallie too is learning all those lessons that most of us take for granted.

Her family say they never expected this transformation, and the speed with which she has improved.When they began their fundraising campaign, it was called 'Hope for Hallie'.They simply wanted to provide a chance of a new life for their daughter.What this surgery has achieved is beyond their wildest dreams and the improvements are continuing.She's begun learning how to walk with a walker and they're looking forward to every new milestone that she makes.

This won't cure her AADC deficiency but they believe this operation has tranformed her quality of life immeasurably.The trial of this gene therapy is still ongoing in Poland but the doctors behind it are hoping in the future this treatment could extend far beyond Hallie's rare condition, and patients with other Parkinson-like disorders could also be helped. And in a world short of positives right now, that is certainly a prospect that is extremely exciting.

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Seven-year-old Hallie Campbell doing well after brain surgery in Poland | ITV News - ITV News

As Savaras cystic fibrosis development program comes to an end, so will the CEO Rob Nevilles 12-year reign at the company.

Following him out the door is his co-founder Taneli Jouhikainen, Savaras president and chief business officer, whos resigning to pursue other opportunities. Matthew Pauls, the former chief of Strongbridge and a member of the board, is stepping in as chairman and interim CEO.

The Austin-based biotech $SVRA still has high faith in molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), and its potential to treat a rare autoimmune lung disorder. A pivotal Phase III trial is planned for the first quarter of 2021.

Last week the company officially shut down an exploratory Phase II trial in CF. After two years of investigating whether the drug can reduce lung infection in adult patients, execs said Vertexs new triple-combination drug plus disruptions due to the Covid-19 pandemic essentially rendered a conclusion impossible.

With Molgradex we have the potential opportunity to bring the first approved therapy for autoimmune pulmonary alveolar proteinosis to market, Pauls said in a statement. We also look forward to disclosing top line results from the Phase 3 AeroVanc AVAIL study early next year and getting regulatory feedback on the Apulmiq clinical development program.

Despite landing breakthrough therapy designation from the FDA, though, molgradex has its share of controversy after missing the primary endpoint and multiple secondary endpoints in an earlier Phase III.

Badrul Chowdhury, a seasoned FDA staffer who moved from AstraZeneca to Savara last November, is staying to steer the drive to market as chief medical officer.

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Savara's long-reigning duo both resign in the wake of CF bust - Endpoints News

About a year after getting the FDA thumbs-up for its blockbuster cystic fibrosis drug Trikafta, Vertex is picking up where it left off.

The Boston-based biotech read out positive results from a Phase III study involving children ages 6 to 11, and announced plans to file for a supplemental NDA later this year. Previously, the triplet regimen was only available to patients 12 and up.

Trikafta, Vertexs $VRTX fourth CF drug, is designed for those with at least one F508del mutation in the CFTR gene. It covers about 90% of CF patients, unlike the companys Kalydeco, which only covers 6% of Americans with CF, according to a STAT report. Within weeks of Trikaftas October 2019 launch, it became Vertexs top-selling drug. Quarterly US sales hit $420 million by the end of January.

The win allowed former CEO Jeffrey Leiden to step down on a high note, passing the torch to current CEO Reshma Kewalramani. Leiden shifted over to executive chairman of the company.

Our aim is to extend eligibility to all patients who may benefit from this transformative medicine, and the positive results from the study in children ages 6 through 11 years old allows us to take another step forward toward this goal, Carmen Bozic, Vertex CMO and EVP of global medicines development and medical affairs, said in a statement.

CF is characterized by the build-up of sticky mucus in the lungs, digestive system and other organs. Its caused by a defective protein that results from mutations in the CFTR gene the most common mutation being F508del. According to Vertex, a Phase III study with 66 children produced safety data consistent with those observed in previous Phase III studies.

The drug cleared primary safety and tolerability endpoints, and showed meaningful improvement across secondary efficacy endpoints, including percent predicted forced expiratory volume in 1 second (ppFEV 1, a marker of cystic fibrosis lung disease progression), sweat chloride, Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score, and body mass index (BMI) through 24 weeks of treatment.

Despite the announcement, Vertexs shares dropped 2.8% on Thursday, closing at $255.65 per share.

In April, the company faced criticism from cost-effectiveness watchdog ICER that its CF drugs were too expensive. Trikafta is priced at more than $311,000 annually, or around $24,000 for a months supply. ICER suggested discounts of up to 77% to match the prices of Vertexs CF drugs with their clinical value.

Despite being transformative therapies, the prices set by the manufacturer costing many millions of dollars over the lifetime of an average patient are out of proportion to their substantial benefits, ICER CMO David Rind said in a statement.When a manufacturer has a monopoly on treatments and is aware that insurers will be unable to refuse coverage, the lack of usual counterbalancing forces can lead to excessive prices, he added later.

Days after Trikaftas FDA approval, AbbVie announced plans to snag a portfolio of CF drugs from Galapagos in a $245 million deal, and bring in a preclinical CFTR potentiator compound from the Cystic Fibrosis Foundation. The Galapagos data looked weak compared to Vertexs, but AbbVie seemed steadfast. It currently has a combo with one potentiator and two corrector molecules in Phase I development.

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A year out from FDA approval, Vertex looks to expand top-selling CF drug to pediatrics - Endpoints News