
Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.
Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.
KYV-101 was developed using the construct Kyverna licensed from the NIH in January 2022.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
NKGen plans to initiate a phase 1 trial pd SNK02 in the first quarter of 2023.
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.
The therapy is currently being evaluated in the phase 1/2 ARYA-1, ARYA-2, and ARYA-3 studies in adults and children.
The first patient has been dosed in a phase 2 study of the allogeneic CAR T-cell therapy GC007g.
In a rodent model, durable auditory brainstem responses to sound were observed after DB-OTO administration.
The director of translational research in myeloma at the Tisch Cancer Institute discussed positive findings, including a 30-month PFS in study participants.
Future data from other participants with LCA enrolled in the phase 1/2 study will be announced at a later date.
The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.
Review top news and interview highlights from the week ending October 14, 2022.
The first patient dosed with IOV-4001 has completed the safety observation period.
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.
The PDUFA target date is set for March 31, 2023.
The chief medical officer of Triumvira Immunologics discussed the company’s future plans.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.
Both botaretigene sparoparvovec and JNJ-81201887 were well-tolerated in treated patients, according to data from the 2022 AAO meeting.
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.
ORR was 80% in the first cohort of phase 1b at the recommended phase 2 dose.
A retrospective study measured correlations of ICANS and NfL in patients with a history of diffuse large B-cell lymphoma.
The director of the Regenerative Medicine Institute at Cedars Sinai discussed results from a phase 1/2a study of CNS10-NPC-GDNF.
Teodora Staeva, PhD, chief scientific officer, Lupus Research Alliance, discussed the potential of the approach in patients with SLE.
Lyell will soon initiate a phase 1 trial of LYL845 with initial data expected in 2024.
Review top news and interview highlights from the week ending October 7, 2022.
The director of translational research in myeloma at the Tisch Cancer Institute discussed investigating sequential T cell redirection therapies.
Interim data from an ongoing study demonstrate continuing efficacy in B-NHL, CLL, and FL.