Gene/cell therapy "reverses" under COVID-19

Guide: In the first half of 2020, regenerative medicine (gene therapy, cell therapy, tissue engineering, etc.) raised $10.7 billion.
the early days of the COVID-19 outbreak, the American Alliance for Regenerative Medicine (ARM) was not sure how the pandemic and the accompanying recession would affect the field of cell and gene therapy.
the answer to that question is becoming clearer as the first half of 2020 draws to a close.
, ARM said in a recent report called "Advanced Innovation InterCOVID-19" that in the first half of 2020, regenerative medicine (gene therapy, cell therapy, tissue engineering, etc.) raised $10.7 billion, surpassing the total funding in 2019 and up 120 percent from the first half of 2019. Janet Lambert, chief executive of
ARM, said: "These surprising funding figures confirm all the enthusiasm for the industry.
I think the drivers of that enthusiasm remain and are optimistic about the second half of 2020.
", of the $10.7 billion, several IPOs were super eye-catching.
include China's CAR-T player Legend Biotech, which made its $487 million Wall Street debut in June.
the same month, gene therapy companies GenerationBio and Akouos raised $230 million and $244 million, respectively.
February, Another gene therapy company, PassageBio, raised $284 million, while gene-editing biotech Beam Therapeutics raised $207 million.
reported that for the first time in the first half of 2020, there will be more than 1,000 developers of regenerative medicine and advanced therapies worldwide.
, 515 are dedicated to gene therapy, 632 cell therapy, and 136 are tissue engineering/biomaterials.
415 of these companies are in clinical development.
is expected to be a record year for regenerative medicine financing despite the economic challenges posed by COVID-19.
healthcare developers raised more money in the first half of 2020 than they did in all of 2019 (H12020 was $10.7 billion, compared with $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 raised by treatment developers for the full year of 2018.
by 2020, almost all financing areas will grow faster than in previous years, with the exception of private/pipeline financing and merger and acquisition financing.
the first half of 2020, there are 1078 clinical trials under way worldwide, including 394 in Phase 1, 587 in Phase 2 and 97 in Phase 3.
in addition, there are 471 clinical trials of cell-based cancer immunotherapy.
notable, there are 11 ongoing clinical trials worldwide using regenerative medicine and advanced therapies to treat COVID-19.
Despite the challenges posed by COVID-19, developers in the field of regenerative medicine and advanced therapies continue to advance clinical programs for various indications to meet medical needs.
listed key development milestones for the first half of 2020: Developers continue to bring late-clinical candidates to market (including China and Japan): On June 9, Mallinckrodt announced that it had completed the submission of a BiologicS License Application (BLA) to the U.S. FDA for the use of Stratatech, a generic human skin replacement product, for adult patients with deep II burns.
May 19, Novartis' AveXis announced that the FDA has approved the use of Zolgensma for the treatment of SMA children with a bistle gene mutation in the motor neuron survival gene 1 (SMN1) under the age of 2. On March 19,
, Zolgensma was approved in Japan.
, April 1, Mesoblast announced that the FDA has accepted a bioproduct license (BLA) application for its allogeneic cell therapy Ryoncil (remestemcel-L) for the treatment of children with steroid refractive acute transplant resistance to host disease (SR-aHD.
FDA also granted priority review of the application and is expected to respond by September 30 this year.
In Japan and China, a sales license for the treatment of B-cell lymphoma has been submitted to Kite/Gilead's Yescarta: On March 30, The First Three filed Yescarta's application in Japan on February 24, Fosun Kate filed Yescarta's application in China on February 13th, Bristol-Myers Squibb) received FDA priority review for its CAR-T therapy liso-cel treatment for adult patients with recurrent or refractable large B-cell lymphoma. On January 28,
, the European Medicines Agency (EMA) approved a marketing authorization application for KTE-X19. on 13 January
, PTCTherapeutics submitted an application for gene therapy PT-AADC to the European Medicines Agency (EMA) for the treatment of aromatic L-amino acid decesase deficiency (AADC).
gene therapy continues to show long-term persistence after several years of treatment: On June 17, bioMarin, a pharmaceutical company, announced the results of an ongoing Phase I/II trial, and data for four years showed that after infusion of experimental gene therapy valoctogeneroaparvovec, the haemorrhage rate of severe haemophiliacs decreased significantly, with a 95% reduction in cumulative average bleeding rate (ABR) and an improvement in THE level of THE VIII factor.
, March 24, Novartis's AveXis announced that data from a long-term follow-up study of Zolgensma's treatment of spinal muscular dystrophy showed that a single dose of Zolgensma is still effective in patients five years after treatment.
all patients treated in the study survived and did not rely on permanent ventilation.
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 (RMAT) qualification.
CTX001 is a research, kinetic gene-edited hematopoietic stem cell therapy that will be used to treat severe sickle cell anemia (SCD) and blood transfusion-dependent beta thalassemia (TDT). On May 6,
, Immunicum announced that Ilixadencel, a cell therapy for kidney cancer, had been granted FDA RMAT eligibility. On April 22,
, Novartis was awarded RMAT status for its CAR-T treatment, Kymriah, for the treatment of follicular lymphoma.
April 16, TissueTech's TTAX02 was qualified by FDARMAT, a candidate for their tissue-based product for intrauterine fetal surgery repair for spina bifida. On 2 March
, MeriaGTx and Janssen's AAV-RPGR Gene Therapy for the treatment of X-chain pigmented retinitis was granted THE EMA Priority Drug Program (PRIME).
, February 27, TESa Therapeutics' CAR-T treatment for recurrent or refractable CD30-positive classic Hodgkin's lymphoma was approved by the FDA for RMAT. on 12 February
, AlloVir's Viralym-M was awarded the PRIME of the EMA, a cell therapy for the treatment or treatment of BK virus, cytomegavirus, human herpes virus type 6, Epstein Bar virus and/or adenovirus for severe infections in immunocompromised patients.
gene editing technology continues to evolve, developers have begun to provide data showing clinical efficacy: on June 12, CRISPRTherapeutics and Vertex Pharmaceuticals presented clinical trial data on THE SELF-sedited hematopoietic stem cell therapy CTX001 based on CRISPR gene editing at the 2020 annual meeting of the European Society of Haematology (EHA).
data show: CTX001 infusion 9 months, the patient did not have VOC, no blood transfusion, total hemoglobin level of 11.8g/dL, fetal hemoglobin 46.1%, F-cell 99.7%.
Allogene Therapeutics and Gracebioll reported preliminary data from clinical trials of their gene-edited allogeneic allogeneic CAR-T therapy: On May 29, Allogene Therapeutics announced allo-501 (isosome) results for recurrent or refractive non-Hodgkin lymphoma: in an ongoing phase 1 study of 19 assessable patients, 37% of patients experienced partial remission.
, April 28, GraceelBio announced the results of a Phase I clinical trial for the treatment of recurrent or refractive (R/R) acute T-lemphoblastic leukemia (T-ALL) by its generic TruUCARGC027 treatment In Phase 1, 5 (100%) subjects received complete remission, including full or incomplete recovery of blood cells (CR/CRi); On March 4,
, Editas and Allergan jointly announced that the first phase I/II clinical trial of CRISPR therapy AGN-151587 (EDIT-101) for leber's congenital black-skinned disease 10 (Lebericamauros10, LCA10) has been completed. On January 8,
, LocusBio launched its first clinical trial of CRISPR-enhanced phage.
conclude, 2020 is a different year.
we've seen a lot of clinical trials of drugs disrupted by the outbreak, 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 for patients with clinical disruption, and regulators have shown a willingness to maintain high health and safety standards while maintaining flexibility.
everything is getting better.
.