Sickle Cell Disease
A page on conditions in our medical research coverage, part of our health reporting, alongside the 28 reports filed on it so far.
ISCHEMIA trial 11-year data confirms no survival gain from invasive strategy
Extended follow-up from the ISCHEMIA and COURAGE trials shows no mortality benefit for an initial invasive strategy over optimal medical therapy...
Beam's risto-cel hits endpoint in sickle cell BEACON trial
Beam Therapeutics' base editing therapy risto-cel achieved its primary endpoint of 12 months without severe pain crises in a key trial, setting up a...
T-Therapeutics secures $133 million for TCR bispecific trials
T-Therapeutics raised $133 million in an oversubscribed Series B to advance its first two TCR-CD3 bispecific programs, TTX-008 for cancer and TTX-018...
FDA Approves First New Head and Neck Cancer Therapy in a Decade
The FDA has granted accelerated approval to Janssen's Rybrevant Faspro for recurrent or metastatic head and neck squamous cell carcinoma, based on a...
UniQure's Huntington's Disease Gene Therapy Gets FDA Priority Review
The FDA has accepted UniQure's application for its Huntington's disease gene therapy ifezuntirgene inilparvovec for priority review.
Combination therapy achieves high major pathological response in pMMR colon cancer
The UNICORN phase 2 trial shows neoadjuvant botensilimab plus balstilimab produced a 36% major pathological response in pMMR colon cancer and 100% in...
FDA Approves Voceryq for Recurrent Respiratory Papillomatosis
The FDA has granted accelerated approval to Voceryq, the first plasmid DNA-based immunotherapy for adults with recurrent respiratory papillomatosis...
Setara Biosciences launches with $90 million for rare disease drug
A new biotech company, Setara Biosciences, has launched with $90 million to develop a therapy for Charcot-Marie-Tooth disease and overactive bladder...
PET-Guided Strategy Enables Chemotherapy De-Escalation in HER2-Positive Breast Cancer
Long-term data from the phase II PHERGain trial shows a PET-guided, response-adapted strategy allowed many patients with HER2-positive early breast...
Industrial Water Pollution Linked to Higher PSP Risk
A 25-year U.K. study finds living near industrial facilities that pollute water with heavy metals and pesticides significantly increases the risk of...
Artiva Biotherapeutics halts AlloNK trial and cuts 94% of staff after patient deaths
Artiva Biotherapeutics is ending development of its AlloNK cell therapy and laying off most employees after two patient deaths in a Phase 2a trial...
Star Therapeutics secures $200 million to refocus pipeline
Star Therapeutics announced a $200 million venture capital financing round on October 23, 2026, to advance its rare disease biologics pipeline...
Camupepimut vaccine shows benefit in pancreatic cancer subgroup
The AMPLIFY-7P phase 2 trial found camupepimut did not improve disease-free survival overall but showed significant benefit in ctDNA-negative...
Estuary Biotherapeutics Launches with $93M for ADPKD Gene Therapies
Estuary Biotherapeutics launched with $93 million in Series A funding to develop one-time gene therapies for autosomal dominant polycystic kidney...
FDA Launches Rare Disease Framework Initiative
The FDA announced the Rare Disease Framework Initiative on October 14, 2026, to create a tailored regulatory pathway for rare disease treatments...
Shionogi Acquires IntraBio for $2 Billion to Expand Rare Disease Portfolio
Shionogi has agreed to acquire biotech firm IntraBio for $2 billion in cash, gaining the marketed rare disease therapy Aqneursa.
EVOKE-03 Trial: Sacituzumab Fails to Boost Survival
The phase III EVOKE-03 trial found adding sacituzumab govitecan to pembrolizumab did not significantly improve survival in untreated metastatic...
GSK's risvutatug rezetecan shows 18.5-month median overall survival
GSK and Hansoh Pharma's antibody-drug conjugate risvutatug rezetecan achieved a median overall survival of 18.5 months in a phase 3 trial for...
Invivyd chairman Marc Elia becomes CEO
Infectious disease biotech Invivyd has appointed board chairman Marc Elia as its new CEO.
Ionis' Zanvastro Wins First FDA Approval for Alexander Disease
The FDA has approved Ionis Pharmaceuticals' Zanvastro, the first treatment for the rare neurological disorder Alexander disease.
FDA approves Ionis’ zilganersen as Zanvastro for Alexander disease
The FDA has approved zilganersen, marketed as Zanvastro, as the first treatment for Alexander disease, a rare and fatal neurodegenerative disorder.
UniQure Seeks FDA Approval for Huntington's Gene Therapy
UniQure has submitted AMT-130, a gene therapy for Huntington's disease, to the FDA for accelerated approval based on three-year data showing it can...
Novartis, BMS Pause CAR-T Autoimmune Trials
Novartis and Bristol Myers Squibb have paused clinical trials of their CAR T-cell therapies for autoimmune diseases following severe adverse events.
Alnylam defends silencer drugs after AstraZeneca trial failure
Alnylam defends its RNAi drugs after AstraZeneca's heart disease trial failure, arguing they work even for patients on stabilizer therapies.