Innovations under COVID-19 "breaking the waves" and gene/cell therapy "reverse currents"

In the early days of COVID-19, the American Alliance for Regenerative Medicine (ARM) wasn't sure how the pandemic and its accompanying recession would affect cell and gene therapy.
as the first half of 2020 draws to a full end, the answer to this question will become clearer.
in a recent report called "Advancing Innovation During COVID-19", ARM said that in the first half of 2020, $10.7 billion was financed in the field of regenerative medicine (gene therapy, cell therapy, tissue engineering, etc.), more than the total funding in 2019, an increase of 120 percent over the first half of 2019.
, chief executive of ARM, said: "These surprising funding figures are proof of all the enthusiasm for the industry today.
I think the drivers of this enthusiasm remain and are optimistic about the second half of 2020.
" specifically, several of the $10.7 billion ipOs are super-eye-catching.
including China's CAR-T race legend Biotech, which led the world in its Wall Street debut in June for $487 million.
same month, gene therapy companies General Bio and Akouos raised $230 million and $244 million, respectively.
February, another gene therapy company, Pasage Bio, raised $284 million, while gene-editing biotech Beam Therapeutics raised $207 million.
2020, for the first time in the world, there will be more than 1,000 developers of regenerative medicine and advanced therapies, according to a report.
, we are committed to the development of 515 gene therapy, 632 cell therapy, tissue engineering / biological materials 136.
415 of these companies are in clinical research and development.
despite the economic challenges posed by COVID-19, 2020 is expected to be a record year for financing regenerative medicine.
healthcare developers raised more money in the first half of 2020 than for the whole of 2019 ($10.7 billion in 2020 and $9.8 billion in 2019).
2018 was the best year on record for cell and gene therapy financing ($13.5 billion), compared with nearly 80 percent of the total financing for the whole of 2018.
by 2020, almost all areas of financing will be growing faster than in previous years, with the exception of private equity/pipeline financing and merger and acquisition financing.
2020, there are 1,078 clinical trials under way worldwide, including 394 in Phase 1, 587 in Phase 2 and 97 in Phase 3.
, there were 471 clinical trials of cell-based cancer immunotherapy.
note that there are 11 ongoing clinical trials worldwide using regenerative medicine and advanced therapies to treat COVID-19.
despite the challenges of COVID-19, developers in the field of regenerative medicine and advanced therapies continue to advance clinical programs for various adaptations to meet medical needs.
The key development milestones for the first half of 2020 are listed here: Developers continue to bring late-clinical candidates to market, including in China and Japan): On June 9, Mallinckrodt announced that it had completed the submission of a biologics license application (BLA) to the FDA to use Stratatech, its generic human skin replacement product, for adult patients with deep II.
May 19 (Xinhua) -- Novarhua's AveXis corporation announced that the FDA has approved Zolgensma for the treatment of children with SMA mutations in the motor neuron survival gene 1 (SMN1) under 2 years of age.
was approved in Japan on March 19.
April 1, Mesoblast announced that the FDA had accepted a biologic licensing application (BLA) for its isotropic cell therapy Ryoncil (remestemcel-L) for the treatment of children with steroid-incurable acute grafts resistant to host disease (SR-aGVHD).
FDA also granted the application priority review eligibility, is expected to respond by September 30 this year.
. In Japan and China, Yescarta has submitted a sales license for the treatment of B-cell lymphoma: on March 30, the first three submitted Yescarta's application in Japan on February 24, Fosun Kate filed Yescarta's application in China. Squibb) received FDA priority review on February 10 for its CAR-T therapy Liso-cel for the treatment of adult patients with recurrent or incurable large B-cell lymphoma.
January 28, the European Medicines Agency (EMA) approved a marketing authorization application for KTE-X19.
January 13, PTC Therapeutics submitted an application to the European Medicines Agency (EMA) for gene therapy PT-AADC for the treatment of aromatic L-amino acid deaceatase deficiency (AADC).
Gene therapy continues to show long-term persistence after several years of treatment: on 17 June, pharmaceutical company BioMarin announced the results of the ongoing Phase I/II trial, with four years of data showing a significant reduction in bleeding rates in patients with severe type A haemophilia after infusion of experimental gene therapy valoctocogene roxaparvoc, with a 95 per cent reduction in cumulative average annual hemorrhage (ABR) and an improvement in VIII factor levels.
March 24 (Xinhua) -- Data from a long-term follow-up study of Zolgensma's treatment of spinal muscular dystrophy showed that a dose of Solgensma was still effective in patients five years after treatment.
all patients treated in the study survived and did not rely on permanent breathing.
FDA and EMA continue to open a "green channel" for rapid approval of regenerative medicine and advanced therapies: On May 11, CRISPR Therapeutics and Vertex Pharmaceuticals announced that the FDA has granted CTX001 Advanced Therapy for Regenerative Medicine (RMAT) status.
CTX001 is a research, self-edited hematopoietic stem cell therapy that will be used to treat severe sickle cell anemia (SCD) and transfusion-dependent beta thalassemia (TDT).
May 6, Immunicum announced that Ilixadencel, a cell therapy used to treat kidney cancer, is eligible for FDA RMAT.
April 22, Novarte qualified for RMAT for its CAR-T treatment for follicle lymphoma, Kymriah.
April 16, TissueTech's TTAX02 qualified for FDA RMAT, their tissue-based candidate for intra-urinal fetal surgical repair for spina bifida.
March 2, Meria GTx and Janssen's AAV-RPGR gene therapy for the treatment of X-chain pigmented retinitis was eligible for the EMA's Priority Drug Program (PRIME).
February 27, Tessa Therapeutics' CAR-T treatment for recurrent or incurable CD30-positive classic Hodgkin's lymphoma qualified for FDA RMAT.
on 12 February, AlloVir's Viralym-M was awarded the PRIME title of EMA, a cell therapy for the treatment or treatment of severe infections of BK virus, cytocytovirus, human herpes virus type 6, Epsteinbar virus and/or adenovirus in patients with impaired immunity.
Gene editing technology continues to evolve and developers have begun to provide data showing clinical efficacy: on June 12, CRISPR Therapeutics and Vertex Pharmaceuticals announced clinical trial data on CTX001, an autobiographical hematopoietic stem cell therapy based on CRISPR gene editing, at the 2020 annual meeting of the European Hematology Association (EHA).
data show that CTX001 infusion 9 months, the patient did not have VOC, no blood transfusion, the total hemoglobin level of 11.8 g/dL, fetal hemoglobin 46.1%, F-cell 99.7%.
. Allogenes and Gracell Bio reported preliminary data on clinical trials of their gene-edited isogenic CAR-T therapy: May 29, Allogene Therapeutics announced ALLO-501 Allogly) for recurrent or incurable non-Hodgkin's lymphoma outcomes: In an ongoing Phase 1 study of 19 assessable patients, 37 percent experienced complete remission and another 26 percent experienced partial remission.
April 28(Xinhua) -- Gracell Bio announced the results of a Phase I clinical trial of its generic TruUCAR GC027 treatment for recurrent or incurable (R/R) acute T lymphoblastic leukemia (T-ALL) : In Phase 1, 5 (100%) subjects received complete remission, including complete or incomplete recovery of blood cell count (CR/CRi), and 4 (80%) subjects received complete remission (MRD-CR) with minimal residual disease negative.
March 4, Editas and Allergan jointly announced that the first patient delivery of phase I/II clinical trials of CRISPR therapy AGN-151587 (EDIT-101) for the treatment of Leber's congenital blackmonia type 10 (Leber congenital blackmonsis 10, LCA 10) has been completed.
January 8, Locus Bio launched the first clinical trial of CRISPR-enhanced phages.
summed up, 2020 is a different year.
we have seen many drug clinical trials interrupted due to outbreaks, and many regenerative medicine companies face challenges in patient recruitment, registration, data collection, and follow-up.
but as outbreak control stabilizes, developers and regulators recognize the potentially disastrous consequences of interrupting clinics, and regulators have shown a willingness to maintain high standards of health and safety while maintaining flexibility.
everything is getting better.
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