Around the Helix: Cell and Gene Therapy Company Updates – July 13, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Amyloidosis Gene Therapy Demonstrates Durable Reductions in Transthyretin
A 93% reduction in serum TTR remained consistent in 6 patients at 6 months.
Legend Biotech Drops Lymphoma CAR T-Cell Therapy
Legend Biotech seeks to prioritize other product candidates in its pipeline.
Gene Therapy May Slow Progression of MPS Type IIIA
Two patients reached significantly higher cognitive development ages than seen in natural history 24 months after dosing.
Duchenne Muscular Dystrophy Gene Therapy Continues to Show Efficacy and Safety
Patients demonstrated a 7-point improvement above pre-treatment baselines on the North Star Ambulatory Assessment at 4 years post-treatment.
Around the Helix: Cell and Gene Therapy Company Updates – July 6, 2022
Gene Therapy for Canavan Disease Shows Early Promise in Safety and Pharmacodynamics
A patient showed more than a 50% reduction in N-acetylaspartate (NAA) in brain white matter at 3 months post-treatment.
Gene Therapy for X-Linked Retinitis Pigmentosa Shows Safety, Efficacy in Phase 1/2 Study
Improvements were reported in retinal function, visual function, and functional vision at 6 months post-treatment.
Around the Helix: Cell and Gene Therapy Company Updates – June 29, 2022
Adrenomyeloneuropathy Natural History Study to Inform Gene Therapy Research
It will be the first AMN clinical study to utilize wearable technology for monitoring patient activity and outcomes.
Gene Therapy for Chronic Pain To Be Explored Under NINDS Grant
Benjamin Harrison, BSc, PhD, assistant professor of biochemistry and nutrition at UNE, shared his thoughts on the research.
Sarepta’s Duchenne Muscular Dystrophy Gene Therapy Placed on Clinical Hold
The hold was related to a serious adverse event of hypomagnesemia in an ongoing phase 2 clinical trial.
Orphan Drug Designation Granted to MB-106 in Waldenstrom Macroglobulinemia
A clinical trial evaluating MB-106 in WM is planned under Mustang Bio's new IND.
Gene-Mediated Immunotherapy for Ovarian Cancer Clears Safety Hurdle, Will Continue Dosing
The phase 1/2 OVATION 2 study is expected to complete enrollment by the third quarter of this year.
Children Treated With Zolgensma for Presymptomatic SMA Achieve Age-Appropriate Motor Milestones
All children in the phase 3 SPR1NT study achieved the primary end point of independent sitting.
Case Study: Neoantigen TCR Gene Therapy Yields Metastatic Pancreatic Cancer Regression
The treated patient achieved an overall partial response of 72% in tumor regression.
NT-I7 Boosts CAR T-Cell Expansion, Persistence, and Anti-Tumor Activity in Preclinical Study
A multisite phase 1 clinical trial for NT-I7 is underway in patients with relapsed/refractory large B-cell lymphoma.
CTX130 Shows Acceptable Safety Profile, Clinically Meaningful Responses in R/R T-Cell Lymphoma
CTX130 is also being investigated for the treatment of r/r renal cell carcinoma in a phase 1 clinical trial called COBALT-RCC (NCT04438083).
Tisa-Cel Shows Durable Efficacy in Pediatric and Young Adult Patients With R/R B-Cell Acute Lymphoblastic Leukemia
Among treated patients, 42% achieved 5-year event free survival.
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