Matt Hoffman

The CAR-T therapy was approved based on the pivotal phase 1b/2 FELIX clinical trial (NCT04404660), which showed good rates of overall complete remission and median duration of remission.

Intellia’s investigational CRISPR-Cas9 gene-editing therapy NTLA-2002 reduced monthly attacks by more than 70% in both tested dose arms.

Patients with melanoma treated with ACTengine IMA203 achieved high response rates, with progression-free survival of 6 months and duration of response beyond 1 year.

Among more than 100 patients with hemophilia A, no FVIII-specific responses showed associations with the assessed safety or efficacy parameters.

The therapy, rAAV-Olig001-ASPA, has shown positive clinical benefits in several interim updates from an ongoing, first-in-human phase 1/2 clinical trial (NCT04833907).

Patients with large B-cell lymphoma in the TRANSFORM trial showed improvements over 3-year period compared with standard of care.

A study of the flu seasons from 2017 to 2020 suggest that cell-based influenza vaccines outperformed egg-based vaccines in effectiveness.

The decision was supported by the phase 3 BENEGENE-2 trial, which suggested that the Pfizer gene therapy was superior to standard of care FIX prophylaxis.

An interim analysis of a phase 1/2 study suggest the REGENXBIO therapy also showed efficacy signals.

A 2-year evaluation of a small cohort of patients with systemic lupus erythematosus, idiopathic inflammatory myositis, and systemic sclerosis showed safety and efficacy which were supportive of additional clinical trials.

Iovance’s tumor infiltrating lymphocyte (TIL) therapy is the first cellular therapy to earn this indication, with the phase 3, confirmatory TILVANCE-301 trial set to verify its clinical benefit.

The ongoing, multistage phase 1/2/3 Cyprus2+ study (NCT04884815) expects to read out initial safety and efficacy data in the first half of 2024.

PepGen’s ongoing multiple ascending dose trial is expected to read out initial data in mid-2024.

These, among many others, are some of the key clinical trials of gene, cell, and regenerative therapies that the CGTLive staff will be following throughout 2024.

The CSL Behring/uniQure gene therapy produced significant reductions in annualized bleeding rates and the number of total bleeds, with a consistent safety profile and no new adverse events.

Among patients with chronic lymphocytic leukemia and small lymphocytic lymphoma, the Bristol Myers Squibb CAR T cell product showed consistent efficacy in patients with a lack of success with BTK inhibitor and venetoclax treatment.

Bluebird bio’s gene therapy, marketed as Zynteglo, showed successful rates of transfusion independence up to 9 years with a reasonable safety profile in data presented at ASH 2023.

The FDA’s Nicole Verdun, MD; and Peter Marks, MD, PhD, offered insight on the recent approvals of exa-cel and lovo-cel, answering questions about the safety of the gene therapies and the continued collection of data on their use.

The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185).

Health Canada has greenlit a protocol amendment for Taysha Gene Therapies’ trial to include patients aged 12 years and older, rather than only adult patients.

uniQure’s AAV9-vector therapy carrying 2 small interfering RNAs targeting GRIK2 was well-tolerated, with a hopeful risk-benefit ratio. A phase 1/2 is set to begin recruitment in late 2023.

The phase 1 VAN-2201 clinical trial (NCT05657301) is assessing the AAV-delivered gene therapy from Chengdu Origen Biotechnology and Vanotech in 5 dose cohorts of 25 total patients.

The CardiAMP autologous cell therapy is being assessed for ischemic heart failure, with a new phase 3 trial that includes modified eligibility requirements and end points.

Eli Lilly’s small interfering RNA therapy showed significant serum reductions with a single dose while remaining well-tolerated. A larger phase 2 study is currently ongoing.

The therapy’s PDUFA date is scheduled for December 8, 2023, and an Advisory Committee is set to meet on October 31, 2023, to discuss the treatment’s potential approval for sickle cell disease.

In a small sample of patients, the CRISPR–Cas9–edited CD34+ hematopoietic stem- and progenitor-cell therapy showed expected safety while inducing red cell fetal hemoglobin.

In a small study, administration of the Généthon therapy encoding UGT1A1 resulted in no serious adverse events. It is also being evaluated in a larger pivotal trial.

XyloCor Therapeutics’ encoberminogene rezmadenovec (XC001) has shown positive data among the 2 highest doses assessed, with the 1×10^11 viral particles dose selected for continued study.

TCR2 Therapeutics’ T cell therapy gavocabtagene autoleucel showed encouraging antitumor activity in the phase 1 portion of an ongoing trial, with an identified recommended dose for the next phase.

The phase 1/2 trial features 3 phases, with new sites expected to open in the coming months. VX-264 uses the same stem cell-derived islets used in the VX-880 program in type 1 diabetes.