Dutch biotech UniQure has won orphan drug designation (ODD) for its investigational treatment for Huntington’s Disease, a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioural abnormalities and cognitive decline.
The National Institute for Health and Care Research (NIHR) and the Medical Research Council (MRC) have published a landscape review report, revealing that £1.1bn has been invested in UK research for rare diseases. The report findings aim to help focus areas for future funding and better target the needs of people with rare diseases. The review found that the NIHR and MRC accounted for almost £627m in funding across 698 studies between 2016 and 2021 for rare disease research. Comprised of 7,000 different types, rare diseases affect around 3.5 million people in the UK. The report revealed that motor neuron disease received the most funding in the portfolio, accounting for 8%, followed by Huntington’s disease (5%) and then preeclampsia (5%). Other diseases that received significant funding included cystic fibrosis (4%), frontotemporal dementia (4%) and idiopathic pulmonary (3%). Over five years, approximately £99.7m out of the nearly £627m of investment was ...
To develop small molecules which can target RNA mis-splicing in neurological diseases, Celgene is launching a $60 million collaboration with Skyhawk Therapeutics. Celgene received the option to develop up to five drug molecules for correcting neurological diseases such as Huntington’s disease, amyotrophic lateral sclerosis etc. The $60 million payment includes milestones, license fees and royalties.
A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute to Reuters on May 15, 2012. REUTERS/National Human Genome Research Institute/Handout.
Don Tracy, Associate Editor Collaboration aims to leverage Nhwa’s expertise in the country’s neuro-psychiatric health sector. Today, Teva announced that it has entered into a collaboration with Nhwa Pharmaceutical Co., Ltd with the goal of providing easier access to Teva’s drug Austedo in China. Austedo focuses on the treatment of neurodegenerative and movement disorders, particularly chorea associated with Huntington disease (HD) and tardive dyskinesia (TD) in adults. According to Teva, the partnership is a part of its goal of expanding the medication as a global brand, as it earned approximately over $1.2 billion in profits last year. Further, Teva reported that Austedo is the first deuterated drug to be approved in China. Deuteration involves replacing hydrogen atoms with deuterium, which can slow down the drug’s metabolism, allowing for less frequent dosing and potentially improving patient compliance.1 “We are committed to addressing the pressing needs of patients in China with more ...
Novartis is paying Voyager Therapeutics $100 million up front to collaborate on gene therapies for Huntington’s disease and spinal muscular atrophy. The deal builds on a relationship the companies started in 2022. By FRANK VINLUAN Novartis is committing $100 million to Voyager Therapeutics to see if the biotech’s technology can lead to new gene therapies for Huntington’s disease and spinal muscular atrophy. According to deal terms announced Tuesday, the sum is an upfront payment that includes a $20 million equity investment in Voyager. Milestone payments could bring the Lexington, Massachusetts-based biotech up to $1.2 billion more. If the partnership leads to commercialized therapies, Voyager would also receive royalties from sales. Gene therapies reach their bodily destinations carried aboard an engineered virus. The therapy’s genetic cargo is enveloped by a protein shell called a capsid. Voyager’s TRACER technology platform discovers capsids that can target particular types of tissue. In addition to ...
Months after the U.S. and European approvals of its CSL-partnered hemophilia A gene therapy Hemgenix, uniQure is selling some royalty rights for up to $400 million. In a deal with HealthCare Royalty (HCRx) and Sagard Healthcare, uniQure agreed to part ways with a portion of its Hemgenix royalty rights. The company previously licensed the drug to Australia’s CSL Behring, and it’s entitled to milestone and royalty payments as the launch progresses. Now, though, it’s trading some of those royalty rights for a quick payday. Under the new deal with HCRx and Sagard Healthcare, uniQure will get $375 million upfront and another $25 million if an undisclosed sales threshold is achieved. The cash lets uniQure “continue to invest” in its adeno-associated virus vector gene therapy pipeline, CEO Matt Kapusta said a statement. The pipeline includes a Huntington’s disease prospect, a refractory temporal lobe epilepsy candidate and an SOD-1 amyotrophic lateral sclerosis ...
After a little over four months as Teva’s CEO, Richard Francis is getting ready to debut his long-term vision for the drugmaker. In Francis’ first quarter as Teva’s CEO, the Israeli pharma giant pulled down $3.7 billion in sales, a flat number versus the same period last year. In Europe, the company posted a 9% sales gain, Teva said Wednesday. Huntington’s disease and tardive dyskinesia med Austedo delivered a 10% global revenue gain to $170 million during the first quarter, while migraine prevention drug Ajovy posted a 35% increase to $95 million. Overall, first-quarter revenues came in around estimates, Barclays analysts wrote in a note to clients. In an investor presentation (PDF), Francis highlighted key attributes of the company, including its innovative pipeline and a “balanced risk portfolio.” The generics group is “a strong business that generates significant cash,” he added on Teva’s first-quarter earnings conference call. But what has ...
The company discontinued about 20 early-stage projects after reviewing their strategic fit and commercial potential. Novartis said Tuesday it will discontinue or license out 10% of its clinical development projects after reviewing their strategic fit and sales potential. The Swiss drugmaker has trimmed its drug pipeline to 136 projects, down by 16 from the 152 disclosed in its fourth quarter earnings report, according to a presentation Tuesday. The biggest cutbacks came to its early-stage projects, with 19 programs cut. Half of those were early-stage programs testing drugs for solid tumors. The move comes one year after Novartis resized its business and narrowed its research focus to five core therapeutic areas — cardiovascular, immunology, neuroscience, solid tumors and hematology. It expects to save at least $1 billion annually by 2024 as a result. “We systematically looked at the pipeline, identified projects that were outside the scope ...
Neurodegenerative diseases are plenty in number, but to find the ultimate research is still ongoing worldwide. One company involved in this process is Suven Life Sciences. They have discovered novel chemical entities responsible for the treatment of neurodegenerative diseases, which were claimed for patenting.
Go to Page Go
your submission has already been received.
OK
Please enter a valid Email address!
Submit
The most relevant industry news & insight will be sent to you every two weeks.