Cell and gene therapy 2020: 5 emerging biopharmaceutical companies to watch

Mumu's emerging small biopharmaceutical companies continue to be at the forefront of innovation, increasingly assuming "responsibility" for drug research and development.
as in a study published by IQVIA called The Changing Landscape of Research and Development, 64% of the 59 new drugs approved by the FDA in 2018 were initially developed by emerging biopharmaceutical (EBP) companies.
companies have worldwide annual sales of less than $500 million and invested less than $200 million in research and development in the past year.
these EBP companies have not only initiated research and development activities for most of the approved new drugs, but have also been able to push 66% of the products under development to the stage of submitting applications for new drugs.
, these emerging small biopharmaceutical companies play an important role in the development of innovative drugs.
Chart 1: Emerging biopharmaceutical companies have patents for nearly two-thirds of the drugs approved in 2018: The Changing Landscape of Research and Development Worldwide, cell and gene therapy (CGT) is changing not only the way humans treat genetic diseases and diseases, but also the entire pharmaceutical ecosystem.
By the end of 2019, more than 27 CGT products will be available worldwide, with about 990 companies engaged in next-generation therapeutic research and development and commercialization, and the global CGT market is expected to exceed $11.96 billion by 2025, according to a Deloitte report.
recently, Biospace analyzed major emerging cell and gene therapy companies to gain a deeper understanding of the entire process from research to clinical applications in this area.
look at five of these emerging cell and gene therapy companies.
1, Abeona Therapeutics Abeona Therapeutics is a biopharmaceutical company dedicated to the development of gene and cell therapies for serious diseases.
's clinical pipeline includes EB-101, an autobiographical gene-corrected cell therapy for recessive malnutrition large herpes looseness, and ABO-102 and ABO-101, a new AAV9-based gene therapy for the treatment of Type A and Type B Sanfilippo syndrome (MPS IIIA and MPS IIIB).
addition, the company's AAV9-based gene therapy portfolio includes ABO-202 and ABO-201 for rare diseases CLN1 disease (infant neuron wax-like lipid lignin deposition or infant barten disease) and CLN3 disease.
currently, the company's EB-101 is conducting Phase III clinical trials, while ABO-102 and ABO-101 are conducting Phase I/II clinical trials.
2, Asklepios BioPharmaceutical Asklepios BioPharmaceutical (AskBio) is a leading AAV gene therapy company headquartered in Chapel Hill, North Carolina.
AskBio's gene therapy platform includes the industry's leading patented cell line manufacturing process Pro10, as well as an extensive AAV case and launcher library.
an early innovator in this field, the company holds more than 500 patents in the fields of AAV production, chimed and self-complementary clothing.
From the research and development pipeline, the company's product line includes a range of products for the neuromuscular disease Pompeia, muscular dystrophy type 2I; central nervous system disease Huntington's disease, Angemann syndrome, Parkinson's disease; cardiovascular disease congesitive heart failure; and metabolic disease methyl dichloremia (MMA).
, AskBio received an international patent application from the World Intellectual Property Organization for the regulation of gene editing systems by the company's founder, Dr. Jude Samulski.
the system took advantage of gene editing while reducing genome-wide off-target effects, immunogenicity, and the challenges of repeated dosing that might arise with CRISPR technology.
is worth noting that Dr. Jude Samulski was the first scientist ever to clone an AAV.
3, AthersysAthersys focus on stem cells and regenerative medicine.
Athersys' leading product, MultiStem, was approved by the FDA in 2012 to treat type I mucosal polysaccharid storing disorder, which is currently being developed to include hemorrhagic stroke, traumatic brain injury, multiple sclerosis, spinal cord injury, congrusive heart failure, and more.
, MultiStem's focus on expanding the adaptation is hemorrhagic stroke, which has entered the FDA's Accelerated Review and Advanced Product Review Channel for Regenerative Medicine.
4, Autolus Therapeutics Autotolus Therapeutics is a biopharmaceutical company that develops the clinical phase of next-generation programmed T-cell therapies for cancer treatments.
autolus has been slowly entering the public eye since 2016, when it raised more than $100 million to develop a new generation of CAR-T therapies.
uses its proprietary T-cell programming technology to engineer precisely targeted, controlled and highly active T-cell therapies to better identify cancer cells, break their defenses and eliminate them.
In April 2019, AUTOTO3, an acute lymphoblastic leukemia (ALL) treatment drug owned by Autolus, was approved by the FDA for orphan drugs, and in November of that year, AUTO1, another drug also used for acute lymphoblastic leukemia (ALL), was approved by the FDA for orphan drugs.
addition to the above two products, the company also has AUTO2, AUTO4, AUTO7 and other product pipelines in the promotion.
5, Editas Medicine Editas Medicine is a genome editing company focused on treating patients with genetic diseases by correcting the genes that cause them.
founders include MIT Professor Zhang Feng, one of CRISPR's pioneers in gene editing technology, who was named one of the top 10 scientific figures in Nature, David Liu, Keith Joung, a Harvard professor, and George Church, a Harvard professor and founder of genetics and molecular biology.
March this year, Editas Medicine and Eljian jointly announced that the CRISPR drug AGN-151587 (EDIT-101) for Leber Congenital Black Haze 10 (LCA10) had completed its first patient The administration, a milestone in AGN-151587 (EDIT-101), the world's first in vivo-delivered CRISPR drug, marks the full launch of its Phase 1/2 clinical trial called BRILLIANCE.
, the company also announced that EDIT-301 as a candidate for gene-editing cell therapy has been awarded a rare pediatric disease (RPD) by the FDA, and EDIT-301 is currently being developed for the treatment of sickle cell disease.
In addition to the EDIT-101 program, Editas Medicine's in vivo gene-editing drugs for the eye include EDIT-102 (treatment of sensorinous neurodeficiability-retinal pigmentation syndrome type 2A) for incoming IND declarations, and a drug for the treatment of normal chromosomal explicit genotype retinitis 4 at the stage of drug discovery.
, the company also has a gene-editing drug to treat Duchy's muscular dystrophy and a treatment for neurological disorders.
conclusion: After more than 30 years of setbacks and precipitation, the field of cell and gene therapy has gradually made many breakthrough research progress.
as more and more companies are stationed in this field, the development of cell and gene therapy in the next decade is worth looking forward to.