[1] The world's first IL-36 antibody was approved for marketing
On September 2, Boehringer Ingelheim, a global biopharmaceutical company, announced that the U.S. FDA has approved Spessolimab (trade name: SPEVIGO) for marketing, becoming the first approved treatment for an adult systemic pustular psoriasis (GPP) attack. Spesolimab is a novel selective antibody that targets IL-36R (interleukin 36 receptor, a key part of the signaling pathway within the immune system) humanized monoclonal antibody that blocks the activation of IL-36R.
According to Boehringer Ingelheim, IL-36 is abundantly expressed in the skin and plays an important role in the pathogenesis of various skin diseases, and they are studying Spesolimab for the treatment of purulent sweat glands and palpital pustular disease (PPP).
• Review: GPP is a rare neutrophilic dermatosis (a group of skin lesions characterized by neutrophil infiltration as a histopathological feature), resulting in general pain, sterile pustules, and serious complications including heart failure, kidney failure, etc. In 2021, Spesolimab has submitted marketing applications in China and Europe, hoping that this new immune drug can benefit patients faster and more.
[2] Novo Nordisk intends to acquire Forma for US$1.1 billion
On September 1, Novo Nordisk, a Danish-based healthcare company, announced that it would acquire U.S. biopharmaceutical company Forma Therapeutics (FMTX.O) for a total value of $1.1 billion (about 7.6 billion yuan) for $20 per share in cash.
Forma was established in 2019 and the deal includes Forma's main development candidate etavopivat, an oral, once-daily, selective pyruvate kinase-R (PKR) activator that is being developed to treat patients with sickle cell anemia (SCD), a hereditary blood anemia.
• Comments: Novo Nordisk was founded in 1923, in the rare fatal blood disease layout is quite a lot, this acquisition of Forma, look forward to the two research and development capabilities, sales capabilities, clinical research and development experience, etc., human beings have a faster breakthrough in sickle cell disease.
[3] Sanofi's first treatment asMD therapy was approved
On August 31, the U.S. FDA announced approval of Sanofi (SNY. Xenpozyme, an innovative enzyme replacement therapy developed by Genzyme, a subsidiary of O) for the treatment of non-central nervous system symptoms in children and adults with acid sphingomyelin deficiency (ASMD), is the first FDA-approved therapy specifically for the treatment of ASMD.
ASMD is a rare progressive genetic disorder in which sphingomyelin lipid accumulation is caused by a lack of enzymes needed to break down complex lipids, and symptoms manifest as enlarged abdomen that can cause pain, vomiting, feeding difficulties, etc. Severe patients will have neurological symptoms and rarely survive beyond 2-3 years of age. According to Sanofi's announcement, Xenpozyme is injected intravenously every two weeks, and the U.S. list price or wholesale price is $7142 per bottle.
• Review: Many rare diseases are life-threatening, but most are still untreated. With the advancement of medical technology, global drug research in the field of rare diseases continues to make progress. The listing of new drugs is not only the hope for patients to survive, but also the hope for the continuation of family life.
[4] Construct the world's first spatio-temporal map of brain regeneration
On September 2, a research team led by Hangzhou Huada Institute of Life Sciences and jointly composed of scientists from 17 units from 3 countries published an article in Science, Suchle-cell Stereo-seq reveals induced progenitor cells involved in axolotl brain regeneration, constructing the first spatio-temporal atlas of salamander brain regeneration. It is also the world's first spatio-temporal atlas of brain regeneration.
The research team studied the laboratory-grown Mexican blunt-mouthed salamander. The Mexican blunt-mouthed salamander can regenerate not only limbs, tails, eyes, skin and liver, but also the brain. Based on BGI space-time omics Stereo-seq technology, the research team systematically analyzed and compared the development and regeneration process of salamander brains, found the key neural stem cell subsets in the regeneration process of salamander brains, depicted the process of reconstructing and damaging neurons in such stem cell subsets, and also found that brain regeneration and development processes have certain similarities.
• Comments: Regenerative repair after brain injury is a major biological and medical problem that needs to be solved urgently. This spatial-temporal map of brain regeneration provides a new direction for the self-repair and regeneration of human tissues and organs, and also provides a valuable data resource for the study of species evolution.
[5] CRISPR gene editing modified CAR-T therapy
On August 31, Nature published a non-viral targeted CAR-T technology (Quikin CART®) collaboration between Shanghai Bangyao Biotechnology Co., Ltd. and East China Normal University and the First Affiliated Hospital of Zhejiang University School of Medicine, "Non-viral, specifically targeted CAR-T cells in B-NHL." Achieve high safety and efficacy in B-NHL), researchers generate non-viruses through CRISPR-Cas9 without using viral vectors, obtaining genome-site integrated CAR-T cell products, providing an innovative technology for CAR-T cell therapy.
In addition, in the "Clinical Trial of PD1 Knockdown Virus-Site Integration CD19-CART Cell Therapy for Relapsed refractory non-Hodgkin lymphoma" conducted in May 2020, the above products demonstrated clinical safety and efficacy, and the first patient to receive treatment has achieved complete disease remission (CR) for more than 2 years.
• Comments: The above research results prove the feasibility of non-viral site-integrated T cell therapy in clinical applications, and this technological innovation also lays the foundation for the development of more gene-targeted modified CAR-T therapies in the future.
[6] Short-term use can obtain a lifelong anti-aging effect
On Aug. 29, Nature Aging published the latest research paper by scientists at the Max Planck Institute for Aging Biology in Germany, "Long-lasting geroprotection from brief rapamycin treatment in early adulthood by increasing intestinal autophagy." Persistently increased intestinal autophagy), the research team developed a short-term regimen for rapamycin that showed similar life-prolonging and age-related lesions in fruit flies and mice to lifelong use of the drug.
The researchers said that the above study reveals new ways to achieve protection in old age through drug intervention, but it is important to determine a clinical dosing timing regimen to maximize protection while reducing side effects; Next, we will investigate whether short-term rapamycin treatment can delay aging in other systems such as cardiovascular and immune systems.
• Comments: Rapamycin, also known as sirolimus and AY-22989, was approved by the US FDA as an immunosuppressant as an immunosuppressant as early as 1999 to reduce rejection after kidney transplantation. With the deepening of scientists' research on rapamycin, a 2009 study demonstrated the anti-aging function of rapamycin. These studies have greatly expanded the understanding of the role of rapamycin as a "life extension drug" at different ages.
[7] AI has designed a new drug type
On August 29, a research team at the University of Washington published a paper in the journal Cell, "Accurate de novo design of membrane-traversing macrocycles," in which the team used an AI platform to design 184 macrocyclic peptides composed of 6-12 amino acids. Of these, 84 designs can cross cell membranes in experimental tests. The researchers say the design method proposed in the study and the macrocyclic peptide molecules capable of crossing cell membranes provide the basis for the development of new oral drugs.
• Comments: In recent years, AI has made continuous breakthroughs in predicting the three-dimensional structure of proteins and synthesizing new proteins, most recently the AlphaFold system developed by DeepMind in July 2022, which predicted 214 million protein structures in more than 1 million species. The above research will further expand the application scope of the AI design platform and open up new avenues for the development of a new generation of oral therapies that accurately target disease biology.
Southern Weekend researcher Luo Xianxian