Advanced 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.
Aucatzyl will look to take market share from Gilead’s Tecartus, and the US approval is a boost for the firm and the wider UK biotech sector.
The drug maker said in its third quarter earnings report that competition from other therapeutic classes, particularly bispecifics, resulted in a slight decline of sales for the CAR-T.
The otherwise encouraging data were marred by one patient death, but the company attributed it to the busulfan conditioning regimen and not BEAM-101.
Lyell believes ImmPACT’s dual CD19/20-targeted CAR-T could offer better response rates than Yescarta or Breyanzi and will focus on IMPT-314 while terminating much of its own pipeline.
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.
The company’s share price has rocketed after becoming the first to show RNA editing works in humans, providing new competition for gene-editing firms.
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.
Emerging Company Profile: The Maryland-based biotech is working on a functional cure for type 1 diabetes that is similar to Vertex’s VX-880 – but with some key differences.
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.
In a video interview with Scrip, Syed Husain, CEO of the US-based cell therapy CDMO BioCentriq, talks about the company’s role in parent GC’s growth strategy and business priorities, the cell and gene therapy manufacturing market and his views on the US BIOSECURE Act.
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.