Advanced Therapies
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.
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: summer ADC dealmaking continues apace; a look at cell therapies in Japan; Pfizer’s new US digital consumer platform; Galapagos’s US point of care CAR-T study; and Novartis partners with Lindy on biologics delivery.
After 10 years of promised investment following its Nobel Prize for iPS cell research, Japan is cautiously narrowing regulations around the conditional approval of cell therapies and cutting some reimbursement prices. Commercial success remains mixed and some products have been withdrawn from the market.
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.
After a quiet July, August and September are shaping up to be busy on the approvals front. Already this month, the US FDA has approved Adaptimmune’s Teclera for synovial sarcoma, Phathom’s Voquezna for gastro-esophageal reflux disease and Servier’s Voranigo for gliomas. Here, Scrip takes a look at ten other approvals for novel products in the offing for the third quarter.
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.