Cell & Gene Weekly
Restauration of profound genetic deafness, improvements in vision for LCA patients, and more...
In continental Europe, it was a short week with some bank holidays - in the spirit of holidays, keeping this one punchy: Enjoy the read and have a good weekend ahead!
Regeneron shares new data for DB-OTO, continuing to show strong efficacy signals
In the ongoing CHORD trial, children with profound genetic deafness received a single intracochlear injection of DB-OTO in one ear, with the other ear serving as a control —> the latest data suggest “dramatical improvments” in hearing, to normal levels in a child (dosed at 11 months of age) and initial hearing improvements in a second child (dosed at 4 years of age) at a 6-week assessment.
“The opportunity of providing the full complexity and spectrum of sound in children born with profound genetic deafness is a phenomenon I did not expect to see in my lifetime, […] With the DB-OTO CHORD trial now enrolling participants in sites across the US, and Europe, we’re part of the beginning of a new era of gene therapy research that looks to create treatment options that address the root cause of profound genetic deafness” Lawrence Lustig - Otolaryngology specialist at Columbia University
As a reminder, DB-OTO was originally developed under a collaboration between Regeneron and Decibel Therapeutics that was established in 2017, with an extension announced in 2021 and the full acquisition of the company last year.
Source: BioSpace; pharmaphorum
Caught My Eye
Editas Medicine - Despite EDIT-101 program in Leber Congenital Amaurosis being discontinued by Editas in Jan 2023 due to a stragegic pipeline re-orgs: a publication from earlier in the weeks seems to suggest relatively strong signals of efficacy in 14 patients (12 adults & 2 children) [link]. Below some of the topline efficacy signals - perhaps it sets up nicely this program for a potential partnership / outliscencing:
11 / 14 patients (79%), showed improvement in at least one of four measured outcomes.
6 / 14 patients (43%), showed improvement in two or more outcomes.
Verve therapeutics - Following termination of the VERVE-101 following SAEs, Verve announced dosing of first patient with the “version 2” of their PCSK9 gene therapy. As a reminder, Verve attributed the side effects of the first gen agent VERVE-101 to the LNP delivery system; as such, VERVE-102 uses a different delivery system than VERVE-101, which includes a different ionizable lipid and Verve’s proprietary GalNAc liver-targeting ligand [link]
Takeda Pharmaceuticals- In their Q1 2024 earning release, Takeda announced the discontinuation of TAK-007, an “off the shelf” CD19-targeted CAR-NK therapy [Takeda earnings presentation]; this is especially noteworthy given last quarter they had announced discontinuation of another cell therapy program, TAK-940 [oncologypipeline]: I believe this leaves them with with just the Allogeneic γδ T cells from the GammaDelta acquisition in 2021. Perhaps a pivot out of cell therapy heme-onc space ?
Quick take news
TScan Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial Evaluating TCR-T Therapy for the Treatment of Solid Tumors [globenewswire.com]
A young patient died due to cardiac arrest after receiving Pfizer's experimental gene therapy being tested in a mid-stage trial for Duchenne muscular dystrophy [Reuters]
AstraZeneca completes equity investment agreement with Cellectis : additional equity investment of $140M [Cellectis IR]
CAR-T cell therapy trial tracker - key themes from this week
~8 new CAR-T studies posted on clinical trials.gov in the past couple of weeks - largely same themes in heme, autoimmune and solid tumors
2 CAR-T studies in autoimmune: (1) Adicet bio CD20 CAR-engineered allo γδ T Cells for Lupus Nephritis [link]; (2) Kyverna Therapeutics CD19 CAR T-Cell for multiple sclerosis [link]
3 CAR-T studies in heme-onc: (1) IIT from Biomédica de Salamanca for a CD19-CAR-T in B cell lymphomas [link]; (2) IIT from Zhejiang University for a Metabolically Armed CD19 CAR-T Cells in B cell malignancies [link]; (3) CARsgen funded trial for CT071 CAR-T in high risk newly diagnosed multiple myeloma patients [link]
2 CAR-T studies in solid tumors: (1) Shanghai Simnova Bio DLL3 CAR-T in SCLC [link]; (2) IIT from Zhejiang University for a CD70 UCAR-T in CD70 expressing solid tumors [link]
Side note: An interesting trial which seems to have started a few years ago, but was only posted last week on clinicaltrials.gov looks into the “Care Pathway and Associated Costs of Patients Treated With CAR T-cells Based on SNDS Data” - courtesy of Novartis [link]: would love to see the results of this one if / when they come out
What I read this week
I was recommended a paper (thank you Connor for sharing), from ICER and NEWDIGS, "Managing the challenges of paying for GTx: strategies for market action and policy reform". Interesting read for the policy and market access folks out there: below a table summarizing some of the “tools” that the paper explores for determinign a fair price for a gene therapies
On X…
“Alyssa is the first patient to receive a base edited therapeutic (triply base edited CAR-T cells that cleared her T-cell leukemia), doing well now more than two years post-treatment thanks to the efforts of Waseem Qasim’s team and GOSH.” [link]
*Any views and opinions expressed herein are those of the author (Marco Sabatini) and do not necessarily reflect those of his employer