CAR T Therapy to be Evaluated in Lupus Nephritis
KYV-101 was developed using the construct Kyverna licensed from the NIH in January 2022.
Around the Helix: Cell and Gene Therapy Company Updates – October 19, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Rigo Garcia, MPH, on Improving DE&I in Rare Disease Health Care
The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
Allogeneic Natural Killer Cell Therapy Given Go-Ahead for Solid Tumors
NKGen plans to initiate a phase 1 trial pd SNK02 in the first quarter of 2023.
Bridging the Gap: Education, Awareness Key to Translating Medical Innovation to Community Health Care in Rare Diseases
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
Clive Svendsen, PhD, on Unlocking the Potential of Astrocyte Cell Therapy
The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.
T-Cell Therapy Recognized for Treating Pediatric Hepatoblastoma
The therapy is currently being evaluated in the phase 1/2 ARYA-1, ARYA-2, and ARYA-3 studies in adults and children.
Gracell Continues Progress in R/R B-Cell Acute Lymphoblastic Leukemia
The first patient has been dosed in a phase 2 study of the allogeneic CAR T-cell therapy GC007g.
Otoferlin-Related Hearing Loss Gene Therapy Gets IND Clearance
In a rodent model, durable auditory brainstem responses to sound were observed after DB-OTO administration.
Samir Parekh, MD, on Improving Survival in Multiple Myeloma With Sequential T-Cell Redirection Therapies
The director of translational research in myeloma at the Tisch Cancer Institute discussed positive findings, including a 30-month PFS in study participants.
Gene Therapy Restores Night Vision in 2 Patients With Leber Congenital Amaurosis
Future data from other participants with LCA enrolled in the phase 1/2 study will be announced at a later date.
Michel Michaelides, MD, FACP, on Gene Therapy's Potential in Retinitis Pigmentosa
The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.
CGTLive’s Weekly Rewind – October 14, 2022
Review top news and interview highlights from the week ending October 14, 2022.
Iovance Advances Another TIL Therapy in Melanoma and NSCLC
The first patient dosed with IOV-4001 has completed the safety observation period.
Anti-HIV CAR-T Therapy Trial Doses First Patient
Preclinical research showed the therapy was able to reduce cellular HIV infection by up to 99% in an in vitro model and more than 97% in a mouse model.
Val-Rox up for Review in Hemophilia A 2 Years After Initial BLA Submission
The PDUFA target date is set for March 31, 2023.
Deyaa Adib, MD, on Optimizing Manufacturing of Solid Tumor Cell Therapies
The chief medical officer of Triumvira Immunologics discussed the company’s future plans.
Around the Helix: Cell and Gene Therapy Company Updates – October 12, 2022
Patients With Fabry Disease Withdrawn From ERT After Isaralgagene Civaparvovec Treatment
All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.
Janssen’s Gene Therapies Show Promise in Retinitis Pigmentosa, Geographic Atrophy
Both botaretigene sparoparvovec and JNJ-81201887 were well-tolerated in treated patients, according to data from the 2022 AAO meeting.
Roberto Giugliani, MD, PhD, on Advantages of Gene Therapy in MPS Type 2
The professor of genetics at Federal University of Rio Grande do Sul discussed how RGX-121 has the potential to change the treatment landscape of MPSII.
TCR-T Therapy Yields Clinical Responses Across Tumor Types
ORR was 80% in the first cohort of phase 1b at the recommended phase 2 dose.
Baseline Neurofilament Light Levels Predict Neurotoxicity With CAR T-Cell Therapy
A retrospective study measured correlations of ICANS and NfL in patients with a history of diffuse large B-cell lymphoma.
Clive Svendsen, PhD, on Astrocyte Progenitor Cell Therapy in ALS
The director of the Regenerative Medicine Institute at Cedars Sinai discussed results from a phase 1/2a study of CNS10-NPC-GDNF.
Achieving Remission in Lupus With CAR T Therapy
Teodora Staeva, PhD, chief scientific officer, Lupus Research Alliance, discussed the potential of the approach in patients with SLE.
TIL Therapy to be Assessed in Relapsed/Refractory Melanoma
Lyell will soon initiate a phase 1 trial of LYL845 with initial data expected in 2024.
CGTLive’s Weekly Rewind – October 7, 2022
Review top news and interview highlights from the week ending October 7, 2022.
Samir Parekh, MD, on Investigating Treatment Options for Relapsed Myeloma
The director of translational research in myeloma at the Tisch Cancer Institute discussed investigating sequential T cell redirection therapies.
New, Multicenter Trial of MB-106 Doses First Patient
Interim data from an ongoing study demonstrate continuing efficacy in B-NHL, CLL, and FL.
CardiAMP Cell Therapy Yields 100% Response Rate in Heart Failure
The new, 2-year data is from a roll-in cohort of 10 patients presented at the HFSA 2022 meeting.
