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Genetically engineered pig liver sustains human life—xeno trial underscores complexity
Surgical teams implanted a genetically modified pig liver into a human recipient and demonstrated that the graft could perform key liver functions for an extended period before complications...
Xenotransplant milestone: Genetically engineered pig liver sustains human
Researchers implanted a genetically modified pig liver into a human patient and demonstrated that the graft could support core liver functions for an extended period before complications forced...
Gene therapies push into children — CRISPR and commercial players aim pediatric expansion
Vertex reported early positive outcomes for its CRISPR-based therapy in children with blood disorders, and ASH briefings signaled broader industry momentum to extend one-time gene therapies into...
Gilead, Arcellx CAR‑T posts deep, durable responses in multiple myeloma
Gilead and partner Arcellx reported pivotal‑stage CAR‑T data showing deep and durable responses in multiple myeloma, with the companies citing high overall response rates and a large proportion of...
MRD as a fast path: New pooled data support surrogate endpoint in AML
A large prospective pooled dataset presented at ASH found that multiparameter flow cytometry–based minimal residual disease (MRD) before consolidation correlates with longer overall survival in...
ctDNA outperforms scans: Natera data show MRD prognostic across lymphoma subtypes
Natera presented real‑world data at ASH showing that circulating tumor DNA (ctDNA) positivity at end of treatment was prognostic across lymphoma subtypes and performed better than PET imaging for...
Clascoterone surprises market — Cosmo stock jumps after baldness Phase III wins
Cosmo Pharmaceuticals released topline results from two identical Phase III trials (SCALP 1 and SCALP 2) for clascoterone 5% solution in androgenetic alopecia and reported statistically...
Axoltis raises $18m to advance BBB‑repair drug for ALS
Axoltis closed an $18 million Series A to advance NX210c, a cyclic peptide derived from an embryonic neuronal protein, through clinical development for amyotrophic lateral sclerosis (ALS). The...
Investor win: Apple Tree Partners prevails in court, secures biotech funding
Apple Tree Partners won a key court decision against an alleged holdout investor, removing a cloud over the firm’s ability to continue financing its portfolio biotechs. The legal victory restores...
Incyte’s calreticulin‑targeted approach shows promise in advanced myelofibrosis
Incyte reported preliminary data showing its experimental agent targeting calreticulin‑mutant myelofibrosis produced meaningful spleen responses and symptom improvements in patients with advanced...
Star Therapeutics readies pivotal programs — Antibody and monthly VWD shot show early promise
Star Therapeutics disclosed early clinical results for two programs: VGA039, an anti‑bleeding antibody with encouraging phase 1/2 data, and a once‑monthly injection for von Willebrand disease that...
Pig liver graft sustains human for days: xenotransplant demonstrates feasibility
Researchers implanted a genetically modified pig liver into a human and showed the graft could support core liver functions for an extended period before complications forced removal. The...
Vertex’s CRISPR therapy hits pediatric milestone – Casgevy shows early efficacy
Vertex reported that its CRISPR‑based therapy (Casgevy/exa‑cel) met early clinical goals in children with sickle cell disease and transfusion‑dependent beta‑thalassemia, signaling potential for...
Gilead’s anito‑cel posts deep responses: next‑gen CAR‑T reads as best‑in‑class
Gilead and partner Arcellx released pivotal‑stage data showing 96% tumor response and 74% complete remission rates for anito‑cel in multiple myeloma, with durable responses and no new safety...
MRD‑negative status predicts survival in AML: pooled data support surrogate use
Researchers presented the largest pooled MRD dataset in acute myeloid leukemia (AML) showing that minimal residual disease (MRD) negativity prior to consolidation correlates with longer overall...
Higher‑dose sickle cell pill raises fetal hemoglobin: Fulcrum reports ASH data
Fulcrum Therapeutics presented early ASH data showing that a higher dose of its oral candidate pociredir induced a clinically meaningful rise in fetal hemoglobin in sickle cell patients. In a...
Exa‑cel shows pediatric benefit: data hint at one‑time functional cure for children
Presentations previewing ASH data indicated exa‑cel (CRISPR/Cas9 gene editing) produced durable benefits in pediatric patients with transfusion‑dependent beta‑thalassemia and sickle cell disease,...
Star’s VGA039 cuts bleeding rates – phase 1/2 drives pivotal dosing and IPO talk
Star Therapeutics reported phase 1/2 data for VGA039, an anti‑bleeding antibody for a common bleeding disorder, showing substantial reductions in bleeding rates after once‑monthly injection. The...
Incyte’s novel myelofibrosis drug shows spleen and symptom benefit – new mechanism targeted
Incyte reported preliminary results from a study targeting calreticulin (CALR) mutations in myelofibrosis, showing meaningful spleen response rates and symptom improvement in advanced disease. The...
Topical clascoterone posts large Phase III gains – Cosmo stock rallies
Cosmo Pharmaceuticals released topline Phase III results for clascoterone 5% topical solution in androgenetic alopecia, reporting a combined 252% relative improvement in Target‑Area Hair Count...