Gene therapy gives new hope for fighting rare disease DMD? Lilly smashed $135 million into the market

Lilly has teamed up with gene editing company Precision BioSciences to unlock the layout of gene therapy for Duchy muscular dystrophy (DMD).
the agreement, Lilly has prepaid more than $100 million and will invest $35 million in Precision's common stock.
, Precision will be eligible for up to $420 million in potential development and commercialization milestones for each product.
, Precision will lead preclinical and IND-supported research, while Lilly will be responsible for clinical development and commercialization.
, DMD has long been the "abyss" of research and development in the eyes of pharmaceutical companies.
lilies, Pfizer had bought Bambo Therapeutics, a gene-editing company that had just financed $50 million, in 2016.
, but it wasn't until May that Pfizer's research and development took a twist and turns to bring good news.
, Lilly has followed suit by investing in DMD gene therapy.
layout DMD, the company is understood to have partnered with Lilly, a gene editing company based in North Carolina, USA, which was founded in 2006.
based on a previous ranking by Duke University, Precision is already among the top private gene editing companies.
2019, Precision is listed on NASDAQ.
while most gene editing companies are now focusing on CRISPR technology, Precision is focusing on ARCUS, a proprietary gene editing technology.
ARCUS derives from a natural genome editing enzyme known as the nesting nuclease, which reprograms an I-Crel gene editing enzyme found in algae, according to its prospectus.
said ARCUS technology has advantages over other gene editing technologies such as CRISPR and TALEN.
' chief executive, matt Kane, called ARCUS "flexible and precise" and avoided the "off-target" effects of other genes that might occur in other technologies.
, the core of ARCUS technology is ARC nuclease, a complete synthase similar to the home nucleic acid intracutase (point-specific DNA cutting enzyme).
ARC nucleases are created using a proprietary set of computer and laboratory techniques to ensure maximum gene editing efficiency and minimal off-target activity.
ALC nucleases are small and highly specific and can be customized to identify DNA sequences within any target gene, according to Precision, a research group.
, based on the analysis of cutting activity in related model organisms, the ability to control potency and specificity can also be achieved by optimizing ARC nucleases.
, Precision also has patent protection for ARCUS technology, unlike other genome editing technologies based on third-party intellectual property.
lilly is working with Precision to develop new gene therapies based on its ARCUS technology.
specifically, the two sides will focus on DMD, as well as two other unresolved genetic targets, using ARCUS for a variety of efficient editing, resulting in a range of therapeutic editing.
note that the partnership also helped Precision, which just lost its eligibility to become a Gilead partner in July.
, Gilead and Precision terminated a $445 million hepatitis B partnership.
, Lilly's vice president of new therapy models, Andrew M. Dr. Andrew C. Adams said the collaboration was another milestone for Lilly in innovative therapies.
Precision will use new treatments, such as gene editing, to address previously difficult targets and adaptive disorders.
"DMD: Research and development in the eyes of pharmaceutical companies "Abyss" Duchy muscular dystrophy (DMD) is a recessive genetic disease of the X chromosome, a rare, irreversible and fatal genetic neuromuscular disease.
incidence of DMD is about 1/3500-1/5000 (newborns) worldwide.
dmD patients generally begin to develop the disease at the age of 3-5 years, the symptoms of the earliest manifestations of aggressive leg muscle weakness, loss of walking ability at 12 years of age, 20 to 30 years of age often due to respiratory failure and death.
so far, there is no cure for this disease in clinical applications.
the Grand View Research report, the global market for DMD drugs is expected to reach $4.11 billion by 2023 and will grow at a compound annual growth rate of 41.3 percent.
but this vast market, but in the eyes of many pharmaceutical companies research and development "abyss".
back in time, many pharmaceutical companies have been in this field of sand.
2016, Novarma's muscular growth inhibitor-targeted therapy bimagrumab (BYM338) failed clinically in Phase III.
pinta745, another candidate drug from Amgen's spin-off company Atara, also failed in a Phase II trial to treat protein energy consumption in patients with end-stage kidney disease.
as early as 2011, Acceleron stopped clinical studies of ACE-031, another muscle growth inhibitor drug used to treat muscular dystrophy, but was abandoned in 2013.
and with gene therapy on the agenda, DMD has new hope of conquering.
lilies, Pfizer bought Bamboo Therapeutics, which had just financed $50 million, in 2016, and Pfizer was looking at Bamboo's gene therapy pipeline.
, four new DMD drugs were rejected by the FDA in six months.
But Pfizer's path to DMD has not been smooth, with clinical trials failing and safety issues breaking out, and it didn't bring good news until May, at a virtual meeting of the American Society for Gene and Cell Therapy's (ASGCT) annual meeting.
data from nine non-bedding (ambulatory) DMD boys show that PF-06939926 intravenous infusion therapy is safe, has encouraging efficacy, and manages safety events, according to the latest clinical data on DMD gene therapy PF-06939926 published by Pfizer.
, the DMD treatment market is only available for treatments such as Sarepta Therapeutics' Exondys 51 (eteplirsen), Vyondys 53 (golodirsen), casimersen, and PTC Therapeutics' Translarna and Emflaza( deflazazat), but the efficacy of these drugs is equally controversial.
advances in gene therapy and the market that remains the Blue Sea are still attracting many players.
addition to Pfizer and lilly, Sarepta, which has two exon jump therapies approved by the FDA, is also working on new gene therapies.
same time, biopharmaceutical company Ultragenyx joined the competition last month in a partnership with Solid Biosciences to develop treatments for Duchma and other diseases caused by a lack of functional muscular dystrophy protein, including Becker muscular dystrophy.
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