CAR-Treg Cell Therapy TX200 in Kidney Transplantation
Sangamo announced dosing of the first patient with a CAR-Treg in their Phase 1/2 STEADFAST clinical study for immune-mediated rejection in HLA-A2 mismatched kidney transplantation from a living donor
What is a CAR-Treg? Lets take a step back: Regulatory T cells (Tregs) play an important role in maintaining immune tolerance. Hence, the idea behind CAR-Tregs is to downregulate regulatory T cells, and thus ‘calm down’ the immune responses. This is a nice first step in trying to expand CAR-T type therapies beyond oncology and provides a nice entry point into Autoimmune space, where a select number of other companies have started to dabble in:
Sonoma bio are the first to the clinic with Treg therapies, looking into their application in indications such as R. Arthritis and T1 Diabetes
Different approach, but also interesting, Cabaletta bio are studying ‘CAARTs’ *not a typo* (Chimeric AutoAntibody Receptor Therapies) for B cell-mediated autoimmune diseases
Source: BusinessWeek
Buzz words of the month: “Reprioritization”
A number of C&G companies are laying off staff and reprioritizing research spending, in what we have come to appreciate as a relatively hostile Biotech market (i.e. public companies have seen a significant downturn: XBI index is ~ -50% vs highs of 2021)
Orchard Therapeutics … discontinuation of a number of programs for rare primary immune deficiencies, including 2 programs that are already in the clinic. Interestingly, they are also discontinuing investment in Strimvelis, which is an EMA approved Gene therapy in collaboration with GSK, but which has had very limited commercial success, having treated only 16 (!!) patients to date in its 6 years on the market…
Taysha GTx…. “one of the fastest seed to IPO biotechs in history”… de-prioritization of a number of programs, including those for GM2 Gangliosidosis, CLN disease, in order to place additional focus on the Giant Axonal Neuropathy (GAN) and Rett Syndrome programs. In the process, they announced a 35% reduction in staff
The list goes on ….
Passage bio: 13 % reduction of workforce and reprioritization of programs
Amicus / ARYA Sciences: 7% reduction in workforce and termination of SPAC merger agreement
Gamida Cell: 10% reduction in workforce and “cutting of planned spending”
Source: Businesswire; BiopharmaDive; Business Journals; Biospace
CAR-Ts continue to gain traction globally with a number of Regulatory approvals
EU: CHMP green light for Novartis’ CAR-T Kymriah in R/R Follicular Lymphoma (3L+) and J&J’s CAR-T Carvykti in R/R Multiple Myeloma (4L+)
Japan: Ministry of Health, Labour and Welfare green light for Breyanzi for 2L treatment of R/R large B-cell lymphoma
Open to suggestions for topics for next weeks’ newsletter:
Vote on menti on next weeks’ topic: https://www.menti.com/fejox4mjyh
Unfolding crisis in Ukraine
With the unfolding humanitarian crisis stemming from the war in Ukraine, I wanted to invite people to continue to show their support by providing donations.
Sleeping bags, medicine, personal hygiene products and baby products are among the most demanded by people on the ground: if you have such products at home, or are able to buy/donate such products, please do check where the nearest collection points are...
Слава Україні, Героям слава