Silas Inman

The CAR T-cell therapy AIC100 demonstrated promising responses and a low level of toxicity in patients with advanced thyroid cancer.

BCMA-directed CAR T-cell therapy anitocabtagene autoleucel demonstrated a 76% complete remission rate and 89% negative minimal residual disease in patients with relapsed/refractory multiple myeloma.

A CRISPR/Cas9-edited allogeneic stem cell transplantation effectively reduced hematopoietic toxicity associated with maintenance gemtuzumab ozogamicin for high-risk acute myeloid leukemia (AML).

CD19-directed CAR-T-cell therapy resulted in sustained remission for over a year and no return of autoimmunity for patients with systemic lupus erythematosus, idiopathic inflammatory myositis, and systemic sclerosis.

Long-term data on lovo-cel gene therapy for sickle cell disease (SCD) reveals sustained efficacy at 60 months.

Patient preferences should be considered when selecting the optimal treatment regimen for patients with relapsed/refractory follicular lymphoma, as both CD19-directed CAR T-cell therapies and CD20-targeted bispecific antibodies can be efficacious.

A final analysis of data from CARTITUDE-1 was also presented at SOHO 2023, which revealed a PFS of 24.9 months in treated patients.

Second-line lisocabtagene maraleucel reduced the risk of an event occurring by 64.4% compared with standard-of-care chemoimmunotherapy induction and autologous stem cell transplantation.

Annualized bleeding rates were superior with the gene therapy fidanacogene elaparvovec compared with factor IX treatment for patients with moderately severe to severe hemophilia B.

The 5-year relapse-free survival rate was 22.3% with T-VEC prior to surgery, compared with 15.2% for surgery alone.

Based on the findings, investigators are considering registration with the FDA and EMA.

Findings from the US-based study align with results from a phase 1b/2 study completed in China of CT041 in patients with gastric and gastroesophageal junction adenocarcinoma.

Updated data at ARVO show that AGTC-501 had sustained efficacy and safety at 18 months for patients with X-linked retinitis pigmentosa caused by RPGR mutations.

SQZ-PBMC-HPV receives FDA Fast Track Designation for patients with advanced or metastatic HPV16+ solid tumors, following promising early results.

Pivotal phase 3 findings for ofra-vec in patients with ovarian cancer are anticipated in the second half of 2022.

NKX101 and NKX019 have demonstrated preliminary signs of safety and efficacy in heavily pretreated patients with AML and NHL in 2 separate phase 1 studies.

KUR-502 elicited promising response rates for patients with relapsed or refractory B-cell malignancies in a phase 1 study.

The FDA has granted a Fast Track Designation to GCC19CART as a potential treatment for adult patients with relapsed and refractory metastatic colorectal cancer.

ADI-001 has been granted a Fast Track Designation by the FDA a potential treatment for patients with relapsed or refractory B-cell non-Hodgkin lymphoma.

The gene therapy ASC618 has received European and US designations and opinions that could help expedite development for hemophilia A.

This designation follows a fast track designation and IND application approval for the agent.

The designation follows the approval of its IND application for the natural killer cell therapy in November 2021.

ADI-001 does not require genetic engineering to remove TCRs to avoid GvHD, making them ideal for an off-the-shelf cell therapy.

A single administration of LN-145 monotherapy resulted in an overall response rate of 21.4% in patients with immunotherapy pretreated metastatic non–small cell lung cancer.

An autologous lentiviral-based gene therapy demonstrated significant benefit, including high overall and event-free survival rates, immune reconstitution, and metabolic correction, for patients with ADA-SCID.

Based on findings from the SPEARHEAD-1 trial, Adaptimmune plans to submit a biologics license application to the FDA in 2022 for afamitresgene autoleucel to treat synovial sarcoma.

The full regulatory approval of agalsidase beta has blocked the accelerated approval pathway for AVR-RD-01 as a treatment for patients with Fabry disease.

Remestemcel-L reduced mortality through 60 days in a prespecified group of ventilator-dependent patients below the age of 65 with COVID-19 and moderate or severe acute respiratory distress syndrome.

A patient enrolled in the phase 2 INFINITY trial exploring ADVM-022 for diabetic macular edema experienced a suspected unexpected serious adverse reaction, which has led to an unmasking of the study.

The FDA has granted an orphan drug designation to the cell therapy ITIL-168 as a potential treatment for patients with stage IIB to IV melanoma.