Cell & Gene Therapy

Cell-based and gene-based therapeutics, including CAR-T, stem-cell therapies, and gene editing.
Cell & Gene Therapy
Biologics

Opus Genetics to unveil OPGx-BEST1 Phase 1/2 data on 9 September

The North Carolina gene therapy company will share Cohort 1 topline results for its BEST1 retinal disease programme at an investor webcast and three ophthalmology
The North Carolina gene therapy company will share Cohort 1 topline results for its BEST1 retinal disease programme at an investor webcast and three ophthalmology
The Biotech Times Editorial Team  · 
Cell & Gene Therapy
Biologics
Regulatory Science

Ultragenyx publishes 96-week Phase 3 data for GENGLYCOS in GSDIa

Peer-reviewed results show a 61% mean reduction in daily cornstarch intake at Week 96, with glycaemic control maintained across both treatment groups.
Peer-reviewed results show a 61% mean reduction in daily cornstarch intake at Week 96, with glycaemic control maintained across both treatment groups.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy
Biologics
Regulatory Science

Ocugen doses first patient in Phase 3 gene therapy trial for dry AMD

Ocugen has initiated its Phase 3 ArMaDa3 trial of OCU410 for geographic atrophy, backed by FDA RMAT designation and a BLA filing targeted for 2028.
Ocugen has initiated its Phase 3 ArMaDa3 trial of OCU410 for geographic atrophy, backed by FDA RMAT designation and a BLA filing targeted for 2028.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

IBA to install Brazil's first proton therapy centre in Rio de Janeiro

IBA has signed a contract with FUSVE to bring proton therapy to Brazil for the first time, with a focus on paediatric cancer care.
IBA has signed a contract with FUSVE to bring proton therapy to Brazil for the first time, with a focus on paediatric cancer care.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Legend Biotech's in vivo CAR-T LB2501 hits 100% ORR in NHL Phase 1

Legend Biotech reported a 100% objective response rate at the higher dose level of LB2501, its in vivo CD19/CD20 dual-targeting CAR-T, without
Legend Biotech reported a 100% objective response rate at the higher dose level of LB2501, its in vivo CD19/CD20 dual-targeting CAR-T, without
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

SL Bio completes SPAC merger, lists on Nasdaq as SL Science

The Taiwan-based cell therapy company closes its Horizon Space SPAC combination, debuting on Nasdaq under ticker SLBT with a $5.6bn implied valuation.
The Taiwan-based cell therapy company closes its Horizon Space SPAC combination, debuting on Nasdaq under ticker SLBT with a $5.6bn implied valuation.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Climb Bio posts Phase 1b ITP data for budoprutug at EHA 2026

Early data from 15 heavily pretreated patients show robust B-cell depletion and durable platelet responses, with no serious adverse events reported.
Early data from 15 heavily pretreated patients show robust B-cell depletion and durable platelet responses, with no serious adverse events reported.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Cullinan Therapeutics reports remissions in SLE and RA with CD19 TCE

Phase 1 data for CLN-978 and velinotamig show clinical remissions and complete renal responses in heavily pre-treated autoimmune patients.
Phase 1 data for CLN-978 and velinotamig show clinical remissions and complete renal responses in heavily pre-treated autoimmune patients.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Cartesian licenses mRNA payload to WestGene for in vivo CAR-T

Cartesian Therapeutics and WestGene Biopharma will combine mRNA CAR-T payloads with targeted LNPs to run a Phase 1 MG trial from late 2026.
Cartesian Therapeutics and WestGene Biopharma will combine mRNA CAR-T payloads with targeted LNPs to run a Phase 1 MG trial from late 2026.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Windward Bio doses first patients in Phase 2 COPD study of WIN378

The Basel biotech has initiated SIRIUS, a Phase 2 COPD trial of its twice-yearly anti-TSLP antibody WIN378, alongside an ongoing asthma programme.
The Basel biotech has initiated SIRIUS, a Phase 2 COPD trial of its twice-yearly anti-TSLP antibody WIN378, alongside an ongoing asthma programme.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

TG Therapeutics launches Phase 2 MG trial after positive BRIUMVI data

Subcutaneous BRIUMVI hit MCID in 82% of myasthenia gravis patients at Week 24, prompting a potentially registration-directed Phase 2 sequential-therapy trial.
Subcutaneous BRIUMVI hit MCID in 82% of myasthenia gravis patients at Week 24, prompting a potentially registration-directed Phase 2 sequential-therapy trial.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy
Regulatory Science

Cellectis wins FDA RMAT designation for allogeneic CAR-T lasme-cel

The Paris-based biotech secured RMAT status for its CD22-targeting off-the-shelf CAR-T candidate in relapsed or refractory B-cell acute lymphoblastic
The Paris-based biotech secured RMAT status for its CD22-targeting off-the-shelf CAR-T candidate in relapsed or refractory B-cell acute lymphoblastic
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Actuate Therapeutics' elraglusib enters BEACON2 neuroblastoma trial

Elraglusib will be assessed alongside dinutuximab beta and chemotherapy in relapsed and refractory paediatric neuroblastoma across up to 60 international sites.
Elraglusib will be assessed alongside dinutuximab beta and chemotherapy in relapsed and refractory paediatric neuroblastoma across up to 60 international sites.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy
Genomics

Sangamo hires Raymond James to explore strategic alternatives

The genomic medicine company is weighing a sale or partnership as it advances a BLA-ready Fabry gene therapy and two CNS programmes.
The genomic medicine company is weighing a sale or partnership as it advances a BLA-ready Fabry gene therapy and two CNS programmes.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Alpha Tau and Tolmar partner to commercialise Alpha DaRT in U.S.

Alpha Tau secures a $35M upfront package from Tolmar, plus up to $161.5M in milestones, for exclusive U.S. prostate cancer rights to its
Alpha Tau secures a $35M upfront package from Tolmar, plus up to $161.5M in milestones, for exclusive U.S. prostate cancer rights to its
The Biotech Times Editorial Team  · 
Cell & Gene Therapy
Biotech

Legend Biotech reports 100% ORR in Phase 1 in vivo CAR-T NHL trial

LB2501, a single-infusion in vivo CAR-T candidate, achieved 100% objective response in six relapsed/refractory NHL patients with no dose-limiting toxicities.
LB2501, a single-infusion in vivo CAR-T candidate, achieved 100% objective response in six relapsed/refractory NHL patients with no dose-limiting toxicities.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

TScan Therapeutics eyes Phase 3 TCR-T launch in Q2 with $128m cash

TScan Therapeutics plans to initiate its pivotal ALLOHA-2 study of TSC-101 in haematological malignancies this quarter, backed by $128m in cash.
TScan Therapeutics plans to initiate its pivotal ALLOHA-2 study of TSC-101 in haematological malignancies this quarter, backed by $128m in cash.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Tempest Therapeutics reports 100% CR rate in dual-targeting CAR-T trials

TPST-2003 achieved complete responses in all 15 CAR-T-naïve evaluable patients across two Phase 1 trials, with Tempest now targeting FDA talks on
TPST-2003 achieved complete responses in all 15 CAR-T-naïve evaluable patients across two Phase 1 trials, with Tempest now targeting FDA talks on
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

IN8bio to present GBM clinical data and TCE platform at ASCO and R&D Day

IN8bio will showcase its gamma-delta T cell engager platform and new glioblastoma clinical data across four conferences in May and June 2026.
IN8bio will showcase its gamma-delta T cell engager platform and new glioblastoma clinical data across four conferences in May and June 2026.
The Biotech Times Editorial Team  · 
Cell & Gene Therapy

Ernexa's ERNA-101 clears tumours completely in ovarian cancer models

Ernexa Therapeutics reported 100% survival and complete tumour clearance for ERNA-101 combined with PD-1 blockade in preclinical ovarian cancer models.
Ernexa Therapeutics reported 100% survival and complete tumour clearance for ERNA-101 combined with PD-1 blockade in preclinical ovarian cancer models.
The Biotech Times Editorial Team  · 
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