sickle cell disease

Agios Pharmaceuticals surged 13% on strong Q1 earnings, with mitapivat revenue jumping 138% to $20.7M. Company plans supplemental FDA application for sickle cell disease expansion.

Fulcrum Therapeutics reports positive pociredir data in sickle cell disease, plans registration trial in H2 2026, maintains $333.3M cash runway through 2029.

Exicure partners with Korea's Adbiotech on co-development of Burixafor for sickle cell disease, leukemia, and solid tumors, targeting IND studies and clinical trials.

Agios Pharmaceuticals plunged 23% after Novo Nordisk's superior sickle cell drug trial results overshadowed Agios' failed mitapavit candidate, threatening its commercial viability.

CRISPR Therapeutics holds $2B cash reserves funding gene-therapy expansion, while Casgevy sales accelerate to $116M in 2025.

Beam Therapeutics CEO John Evans sold 30,078 shares for $739K via pre-planned tax obligation. Strong 2025 revenue growth and $1.2B cash reserves indicate company health.

Agios Pharmaceuticals to report Q1 2026 results April 29 amid FDA accelerated approval pursuit for sickle cell mitapivat and UAE thalassemia approval.

Agios Pharmaceuticals surges on FDA accelerated approval for mitapivat sickle cell treatment, enabling faster path to full approval despite company's ongoing losses.

MedicAlert Foundation, SCDAA, and Fulcrum Therapeutics launch three-year partnership to improve emergency department access to sickle cell patients' critical care information via smart medical ID cards.

Fulcrum's pociredir shows strong fetal hemoglobin gains in sickle cell Phase 1b trial, with 58% of patients reaching therapeutic levels and reduced pain crises.

Beam Therapeutics secures $500M credit facility from Sixth Street Partners to fund commercial launch of its sickle cell therapy, with payments tied to clinical and regulatory milestones.

CRISPR Therapeutics eyes growth from Casgevy commercialization and pipeline expansion. FDA-approved gene therapy generates revenue while multiple clinical programs advance toward 2026 milestones.

CRISPR Therapeutics reports strong Q4 results with Casgevy gene therapy generating $116M revenue and 147 patient initiations, signaling accelerating adoption and market confidence.