Reg Med - MåsteLäsning

Branschorganisationen Alliance for Regenerative Medicine har sammanställt ett faktaunderlag som i allra högsta grad berör tillverkare av utrustning till det starkt växande området Reg Med. De har identifierat vilka områden inom Reg Med som kommer växa snabbast och bli störst. Av den anledningen bör instrumenttillverkare och dito annan utrustning ha beredskap på en förmodat kommande stor efterfrågan med synnerligen bra säljmöjligheter. 

"Science is moving at a rapid pace, creating technologies that could provide immense benefits to patients. Our new horizon scan identifies several groundbreaking approaches poised to change the industry and shape the future of biotechnology."

I denna 52-sidiga faktasamling får branschens aktörer ovärderlig information om Reg Meds framtid. Broschyren bör ligga på alla inblandade VD;ars skrivbord och gärna hemma på nattduksbordet vid sängen så den lusläses flera gånger om. Ok,men till innehållet i ett sammandrag. Först en kort inledning:

Methodology for Horizon Scan of Emerging Technologies 

 There are many ongoing efforts seeking to enhance the efficacy and safety of available strategies. However, this whitepaper, based on a recently completed horizon scan, seeks to identify emerging technologies in Cell and Gene Therapies (C&GTs). Prior to initiating the horizon scan, a team from Catalyst Healthcare Consulting and Alliance for Regenerative Medicine worked to create an unbiased approach to scan the C&GT industry. This approach began with defining “emerging technologies” as innovative technologies with substantial untapped clinical potential, capable of shaping the future of the biotech industry over the next 10 years. The horizon scan included any technology that will be regulated by CBER OTP and can be directly translated into or enable the translation of a marketed product.

Analytikerna har utgått från specifika kriterier och gjort en 10-1-topp lista på vilka områden inom Reg Med som kommer växa snabbt och bli störst.

Branschens alla aktörer som i någon form är involverade i T-Cell  och CAR T-Cell ska omedelbums frigöra kapital och sätta igång att masstillverka sin utrustning,sina instrument. Det så de inte står med tomma lager när det stora brejket kommer. Den försäljningsutveckling dessa aktörer sannolikt kommer ha lär de inte ha upplevt tidigare.

Vidare i analysen :

01 Background 

 Systemic autoimmune diseases are primarily caused by the production of autoantibodies or autoreactive cytotoxic T lymphocytes (CTLs), both of which recognize self-antigens as foreign and result in an excessive, damaging immune response. Autoantibodies, which are produced by self-reactive B cells, bind to self-cells in the body and mediate their destruction. Similarly, autoreactive CTLs use their T cell receptors (TCRs) to interact with major histocompatibility complex class I (MHC-I) molecules on the surface of self-cells, resulting in apoptotic death. Cell death mediated by these self-reactive immune cells results in widespread inflammation that can ultimately damage tissue, among other severe health complications.Current treatments for autoimmune diseases, such as lupus, multiple sclerosis, inflammatory bowel disease (IBD), and more, rely largely on systemic immunosuppressant medications. These non-specific treatment regimens are associated with nausea and vomiting, fatigue, and increased risk of infection, facilitating the need for alternative treatment strategies, such as T cell-based therapies.There are three major subsets of T cell-based therapies currently in development to target an imbalanced or hyperactive immune system associated with autoimmunity: CAR-T cell therapies for autoimmunity, engineered regulatory T cell (Treg) therapies, and Treg transplants.Given their efficacy in recognizing cancer-related antigens and destroying tumor cells, CAR-T cell therapies are an established platform that is currently being leveraged for autoimmunity applications. All approved CAR-T cell therapies are designed to target antigens associated with proliferative B cells (CD19 or BCMA), achieving success in targeting liquid cancers caused by B cell permutations (e.g., leukemias, lymphomas, and myelomas).Since some autoimmune diseases are characterized by an increase in autoantibodies produced by dysfunctional B cells, CD19- and BCMA-targeting CAR-T cells may be repurposed to reduce autoantibody production by mediating B cell killing. Tregs are a heterogenous population of immune regulatory cells that can help reduce the killing activity of CTLs to prevent a hyperactive immune response. Tregs are a subset of CD4+ T cells that are characterized by their co-expression of CD25 and FOXP3, the latter of which is an essential transcription factor for mediating their immunosuppressive activity.Polyclonal Treg therapy – in which Tregs are isolated from a patient, expanded in culture, and reinfused into the patient– has had mixed results in terms of therapeutic efficacy for autoimmune disease patients.Therefore, cell therapy developers have begun using Tregs as a candidate cell type for gene modified cell therapy.

02 Areas of active therapeutic development

CAR-T cell therapies for autoimmunity currently in development seek to target CTLs to hyperactive, autoantibody-producing B cells. To do so, CARs are engineered to express the autoantigen itself, facilitating the binding of CAR-T cells to the autoantibodies on B cells to mediate their destruction . Outside of traditional CAR-T cell approaches, developers have also begun engineering Tregs to express CARs or TCRs to direct their immunosuppressive activity to sites of autoimmune disease. For example, Tregs have been engineered to induce immunosuppression at sites of self-reactive lymphocyte-mediated cellular destruction through the expression of TCRs or CARs that recognize antigens on the cells that are being destroyed. For example, TCR-Treg cells engineered to recognize pancreatic islet cells are being developed to suppress their destruction by self-reactive CTLs for Type I diabetes indications.Moreover, TCR-Tregs have been designed to hone to and mediate immunosuppression at sites of myelin accumulation, the underlying cause of progressive multiple sclerosis, thus reducing disease severity.

 As an alternative mechanism to directly recognizing antigens on cells likely to be destroyed by self-reactive T cells, CAR-Tregs have also been engineered to recognize inflammatory cells that contribute to autoimmune severity. For example, Tregs engineered with CARs recognizing IL-23, a receptor that is overexpressed by inflammatory cells in the GI tract of Crohn’s disease patients, have been shown to reduce local inflammation associated with disease.Similarly, CAR-Tregs that recognize certain classes of HLA molecules present on newly transplanted organs are being developed to suppress the immune reactivity to these foreign antigens to reduce the risk of graft-vs-host disease (GvHD).Finally, cell therapy developers are also optimizing enrichment protocols to select and expand for allogeneic, non-engineered Tregs from healthy cord blood that can naturally hone to sites of inflammation and mediate immunosuppression for a variety of disease indications.

Resten av analysen får ni läsa själva,ovanstående urval är de viktigaste delarna enligt mig.

Ok,då är vi tillbaka till mitt favoritområde igen. T-Cell och CAR T-Cell. Nu kanske alla förstår vad som kommer hända inom dessa områden.Med risk för att bli tjatig upprepar jag mig med att säga : HoloMonitor har sin givna plats inom dessa 2 snabbt växande områden och PHI kan ha en räkmacka in i denna kommande förmodat sanslöst stora bransch,givet att de är medvetna om vad som serveras framför dem.

Jag tar mig friheten till upprepning en sista gång. 😎

Branschens alla aktörer som i någon form är involverade i T-Cell  och CAR T-Cell ska omedelbums frigöra kapital och sätta igång att masstillverka sin utrustning,sina instrument. Det så de inte står med tomma lager när det stora brejket kommer. Den försäljningsutveckling dessa aktörer sannolikt kommer ha lär de inte ha upplevt tidigare."

Ps.Vad tror ni det skulle innebära för PHI om de fick in en fot i 1 eller bägge dessa nya områden?
Fundera på det under helgen och säg sen vad aktien egentligen borde kosta.

                                                Mvh the99