Cell & Gene Weekly
Galapagos and Blood Centers of America pact for decentralized manuf, and more...
Apart from the earnings, a relatively quiet week in the cell and gene therapy space. Have a good weekend all !
Caught My Eye
Commercial differentiation via decentralized manufacturing network for new CAR-T players: Galapagos and Blood Centers of America are partnering to try to establish a nationwide network of decentralized CAR-T manufacturing in the U.S.. An interesting concept that could give them an advantage vs the currently entrenched marketed CAR-Ts, *should Galapagos receive approval from the FDA for their CAR-T therapies.
The partnership, will give Galapagos access to the BCA’s community blood centers —> manufacturing will be closer + apheresis capacity expanded + BCA will leverage its footprint to support site initiation / onboarding: SMART ! Ultimately, the the idea is to achieve treatments in ~1 week timeframe, which would be a significant improvement over current competition (~2-3 weeks) [source; source]Functional cure for cold sores / herpes? Data for an experiemnetal gene therapy showed reduced viral load and shedding in mouse models - published in Nature Comms, this experimental therapy eliminated 90% of herpes simplex virus 1 (HSV-1) after facial infection, and 97% of herpes HSV-1 after genital infection in mice [source]
Earnings Season - status check
Status check on commercialization of Cell and Gene therapies (other than CAR-Ts given I covered these a couple of weeks back in my newsletter)
Gene therapiy sales Q1 2024 are edging towards $500M in combined sales, predominantly driven by Zolgensma and Elevidys
Zolgensma $295 M- with current label, Novartis has seemingly reached a plateau, at least until the IT formulation comes in
Elevidys $134 M- despite a relatively narrow label and a relatively small increase QoQ, Elevidys has been able to generate significant sales in just 3 quarters; as stated in the earnings release, cumulative revenue “far exceeds performance of all other gene therapies approved in the last few years combined” [not that there were that many to compare to 😉 ]
Vyjuvek $45 M - continued momentum for Krystal in their commercialization of their topical gene therapy for DEP (note - following a CRL for Krystals competitor, pz-cel, Abeona path to commercialization has been significantly delayed - earliest FDA approval now in 1H 2025)
Bluebird bio $18.6 M across their three CGTs- Limited details given they notified investors that the 10-Q (where all the interesting details are!) will be submitted late - the $18.6 M is combined earnings for their 3 therapies, mainly driven by Zynteglo currently
Sources: Sarepta, Bluebird; Krystal
CAR-T cell therapy trial tracker - key themes from this week
~6 new CAR-T studies posted on clinical trials.gov in the past week - some important trials in the heme space, notably, a large trial (n=450) from Kite / Arcellx for a BCMA CAR-T in 2L+ MM (H2H trial vs SoC)
2 CAR-T studies in autoimmune: (1) PersonGen CD19 CAR-T in R/R Autoimmmune [link]; (2) Liangjing Lu CD19 CAR-T for treatment of systemic sclerosis [link]
3 CAR-T studies in heme-onc: (1) Kite / Arcellx ddBCMA CAR-T compared to SoC (poma, bortezomib, dexa, dara, etc.) in R/R MM [link]; (2) Stanford Uni study for CD22 CAR T usage post commercial CD19 [link]; (3) GPRC5D CAR-T from Carsgen in newly diagnosed MM [link]
1 CAR-T studies in solid tumors: (1) Beijing Immunochina Medical IM83 CAR-T in t R/R osteosarcoma [link]
Interesting side note: One clinical trial this week was initiated to evaluate vital sign monitoring devices, TempTraq and VitalTraq to promote earlier detection and intervention of treatment-related toxicities from CAR-T use, including cytokine release syndrome (CRS) and febrile neutropenia. [link]
*Any views and opinions expressed herein are those of the author (Marco Sabatini) and do not necessarily reflect those of his employer