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Path Ahead For Neuraly’s NLY01 Unclear After Phase II Parkinson’s Disappointment
The US firm’s lead program has flunked a mid-stage trial in early Parkinson’s disease and, despite a promising efficacy signal in a younger patient cohort, its future remains uncertain.
AstraZeneca And Ionis ATTR Ambitions Boosted
The companies are hoping that new data on recently-filed eplontersen will boost the drug's chances of challenging Alnylam’s Amvuttra and Onpattro.
Quick Listen: Scrip's Five Must-Know Things
In this week's podcast version of Five Must-Know Things: ripples from SVB’s failure; Karuna’s schizophrenia win; big pharma remains open to partnering; ChatGPT comes to medical congresses; and China’s COVID vaccine lessons.
Novartis’s ‘Poster Child’ Zolgensma Shows What Gene Therapy Can Do – And Its Limitations
The clinical development head of Novartis Gene Therapies talks about the progress for SMA patients more than seven years after treatment began, and hopes of boosting outcomes by treating pre-symptomatic patients.
PureTech Cashes In On Promise Of Karuna Schizophrenia Drug
Karuna’s KarXT could be the first new mechanism for treating schizophrenia approved in more than 50 years and founder PureTech is tapping into that promise by linking up with Royalty Pharma and boosting its cashpile.
Biohaven Makes Bet On Brain-Penetrant TYK2/JAK1 Inhibitor For Neurological Diseases
The company, which has struggled in the clinic following a deal where Pfizer bought its migraine portfolio, is licensing the dual inhibitor drug from a Chinese firm for up to $970m.
Long-Term Data Boost Novartis And Roche SMA Challenge To Biogen’s Spinraza
New long-term data from Novartis’s Zolgensma and Roche’s Erysdi will help close the gap on Biogen’s market leader Spinraza, but the three therapies look set to develop distinct or even complementary roles as the market matures.
Double Blow For Sangamo’s Zinc Finger Approach As Novartis And Biogen Deals Collapse
The US biotech is facing yet another setback on its journey to proof-of-concept for its proprietary gene editing technology as two pharma firms back out of collaborations in the neurological space.
Karuna Three-For-Three As Another KarXT Schizophrenia Trial Hits Primary Endpoint
The company is on track for a mid-2023 FDA filing, while executives suggested that they would likely pursue partnership for commercialization of the drug in Europe.
Obstacle Thrown In Sarepta’s Path To Market As Gene Therapy Will Face AdComm After All
The route to market for Sarepta’s gene therapy candidate SRP-9001 for Duchenne muscular dystrophy has just got more complex after a U-turn by the US FDA. Its accelerated approval will now be scrutinized by outside experts – but the company remains confident in its data.
Scrip Asks…What Does 2023 Hold For Biopharma? Part 8: Therapeutic Area Advances
Neuroscience and oncology fuel high hopes for therapeutic R&D progress among 90-plus surveyed biopharma industry leaders.
‘Epigenetic Engineering’ Gene Therapy From Epic Bio Shows Early Promise
The San Francisco-based company has taken a step forward with proof of concept for its ‘switch on or switch off’ approach to gene regulation via non-cutting CRISPR molecules.
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