Around the Helix: Cell and Gene Therapy Company Updates – December 7, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
BioCardia Follows up CardiAMP With CardiALLO Clearance for Heart Failure
The allogeneic mesenchymal stem cell therapy trial will enroll patients ineligible for treatment with the autologous CardiAMP.
Genome-Edited Cell Therapy Reduces VOEs in Sickle Cell Disease
The first 2 participants dosed are free of vaso-occlusive events as of 5 and 1.5 months of follow-up after treatment with EDIT-301.
Leveraging Cell Therapy Advances for Treating Hodgkin Lymphoma, Other Cancers
Ivan Horak, MD, discussed Tessa’s cell therapy platforms and the ACTION clinical trial.
Neural Cell Therapy Reduce Seizures in MTLE
Updated data were presented at the AES annual meeting in Nashville.
Nkarta's Allogeneic CAR T Shows Clinical Responses in Non-Hodgkin Lymphoma
Patients with non-Hodgkin lymphoma in Nkarta's phase 1 study had a 75% complete response rate.
Nirav N. Shah, MD, on Unmet Needs With CAR T-Cell Therapies in Hematologic Malignancies
The associate professor from the Medical College of Wisconsin discussed how to address unmet needs with CAR T-cell therapies.
Retinitis Pigmentosa Gene Therapy Cleared for Trials
Initial safety data from the PRODYGY study are expected in 2023.
Multiplex-Base Edited CAR T Therapy Cleared for Trial in T-ALL After Hold
Beam Therapeutics provided data on genomic rearrangement, off-target edits and from a cytokine independent growth assay to the FDA in November 2022.
CGTLive’s Weekly Rewind – December 2, 2022
Review top news and interview highlights from the week ending December 2, 2022.
Allogene’s CAR T Therapies Show Antitumor Activity in Solid and Liquid Tumors
Updated data from 3 of the company’s programs were presented at an R&D showcase.
Leber Congenital Amaurosis 5 Gene Therapy Cleared for Trials
OPGx-001 is the first of Opus Genetics’ gene therapy candidates for treating LCA to enter clinical trials.
OTOF Hearing Loss Gene Therapy Granted Orphan Drug Designation
Sensorion indicated it is still on track to submit a clinical trial application in the first half of next year.
Arshad Khanani, MD, on Reducing Treatment Burden in Wet AMD With Gene Therapy
The director of clinical research at Sierra Eye Associates discussed unmet needs in patients with wet AMD.
Around the Helix: Cell and Gene Therapy Company Updates – November 30, 2022
GM1 Gangliosidosis Gene Therapy Trial Safe to Continue
Lysogene’s MPS IIIA gene therapy program recently failed its primary endpoint in the phase 2/3 AAVANCE trial.
Etranacogene Dezaparvovec Approval May Pave Way for Val-Rox in Hemophilia A
The FDA has decided not to hold an advisory committee meeting for Roctavian after all.
FDA Recognizes Allogeneic CAR T Therapy for Lymphomas
CB-010 most recently demonstrated a 100% complete response rate in data presented at the EHA 2022 congress.
Base-Editing HSC Therapy Enrolls First Patient With Sickle Cell Disease
BEAM-101 is edited with a next-generation CRIPSR product to mimic single nucleotide polymorphisms that enable persistence of fetal hemoglobin.
ARV Cardiomyopathy Gene Therapy Gets Orphan Drug Designation
In preclinical research, TN-401 was shown to significantly increase the lifespan of PKP2-knock-out mice.
Sarepta’s DMD Gene Therapy Accepted for Priority Review
SRP-9001's PDUFA date is set for May 23, 2023.
CGTLive’s Weekly Rewind – November 25, 2022
Review top news and interview highlights from the week ending November 25, 2022.
NRTX-1001 Reduces Mesial Temporal Lobe Epilepsy Seizures in 2 Patients
The first participant has been followed-up for 3 months so far after treatment while the second is only 1 week post-treatment.
Around the Helix: Cell and Gene Therapy Company Updates – November 23, 2022
Etranacogene Dezaparvovec, Marketed as Hemgenix, Is First Gene Therapy Approved for Hemophilia B
A marketing authorization application for etranacogene dezaparvovec is currently under review by the EMA.
Legend Biotech Brings SCLC CAR T to Trials
The IND clearance comes after Legend terminated another clinical trial in T-cell lymphoma in July 2022.
Targeting Hematological Malignancies With Autologous and Allogeneic Cell Therapies
Thomas Willemsen, president and chief executive officer, Tessa Therapeutics, discussed the company’s technologies and 2023 milestones.
CaMMouflage Study to Evaluate Allogeneic Anti-BCMA CAR T Therapy in Multiple Myeloma
The phase 1 clinical trial plans to initiate enrollment in early 2023.
Gene Therapy to be Assessed in Heart Failure With Preserved Ejection Fraction Trial
SRD-001 is designed to increase expression and functional activity of SERCA2a, down-regulation of which is associated with all forms of heart failure.
New Afami-Cel Data to Support BLA Submission in Synovial Sarcoma
Updated data from the SPEARHEAD-1 study were presented at the 2022 CTOS Annual Meeting.
