Gene Therapies
The company is trading below cash after admitting that an adverse event in a Rett syndrome trial left a patient critically ill.
Following ex-US approvals, PTC obtains a US OK for Kebilidi, branded Upstaza outside the US, as a gene therapy for ultra-rare AADC deficiency. The drug is administered via neurological surgery.
The biotech anticipates treating 74 patients with its gene therapies this year, with 30 more already scheduled for 2025.
Analysts also looked into how much differentiation there was between data for the gene therapy NGN-401 and what Taysha has shown with TSHA-102 in the same disease.
The otherwise encouraging data were marred by one patient death, but the company attributed it to the busulfan conditioning regimen and not BEAM-101.
The gene-editing therapy produced functional cures in eight of 11 patients receiving the go-forward 50mg dose, but the rate fell short of what some investors had hoped for.
Multiple Japanese ventures presented updates on their novel pipeline assets and modalities at BioJapan, including cell and gene therapies and combination treatments, despite lingering financing challenges.
Ocuphire will shift toward gene therapy for inherited retinal degeneration via a merger that will take Opus Genetics public. The firm will also be responsible for one of Ocuphire’s pipeline projects.
ARM’s Cell and Gene Meeting on the Mesa offered optimism that a biopharma financial market recovery is under way, but cell and gene therapies still are seen as risky investments.
Big pharma executives at ARM’s Cell and Gene Meeting on the Mesa shared some of the practical challenges of bringing one-time treatments to market in hemophilia, cancer and beyond.
With more advanced therapies gaining approval, companies at ARM’s Cell and Gene Meeting on the Mesa considered hurdles to making their products more broadly available and developing them for larger indications.
Purespring has just raised $105m from an investor syndicate who are betting it can develop the first gene therapy for kidney disease and learn from setbacks experienced in the space.
An upfront payment of $110m from BMS has come just in time for Prime Medicine, but its real test will be the first Phase I data, expected early next year.
The company said the results did not meet the threshold for continued capital investment, giving a boost to Spruce Biosciences and its competing congenital adrenal hyperplasia program.
CorrectSequence led the pack of Chinese cell and gene therapy developers seeking new funding with a roughly $14m series A-plus round. In other modalities, antibody-focused developers Mabgeek and Novamab closed series Bs.
In this week's podcast edition of Five Must-Know Things: Merck & Co. steps into CD19 bispecific space; gene therapy patients rise, but slowly; Madrigal’s Rezdiffra plans; Korean biopharma financing recovering?; and approvals to watch out for in Q3.
Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area.
The company will cut 65% of its workforce to extend its cash runway and focus its resources on AMT-130 for Huntington's disease and three earlier-stage programs moving into Phase I/II studies.
The company is hoping to become second to market with a DMD gene therapy after Sarepta’s Elevidys, which recently had a label expansion.
The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.