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Gene therapy, an extremely cutting-edge technology, is at the cusp, and many people want to stand on the cusp but are worried about unknown risks, so they stop and wait and look for suitable opportunities
.
In 2022, gene therapy has reached a new level in twists and turns, with a total of 5 drugs approved by the FDA and EMA, the most approved gene therapy drugs in history
.
Previously, two drugs, Luxturna and Zolgensma, were approved to catalyze the gene therapy market, and large pharmaceutical companies began to acquire and cooperate to lay out gene therapy
.
Gene editing companies were born and entered the clinic, with a market value of more than 10 billion US dollars
.
For a new technology, validation over time increases certainty and risk decreases
.
So what will happen to gene therapy next year? Can you find the right investment opportunity?
In 2023, challenges at all stages of foreign gene therapy will hit, and gene editing will attract attention; Domestic gene therapy has entered its infancy and will carry out a large number of clinical trials
next year.
Now we will sort out the key nodes in the field of gene therapy in 2023 and find the right time
.
1 Commercialization nodeIf the road of commercialization can continue to be smooth, and the last hurdle is opened, more and more people will enter the gene therapy and usher in a new boom
.
.
This year, 5 gene therapy drugs were approved, specific to each product, these drugs will face commercialization problems, and sales and expectations or expectations will directly affect the stock price
.
And these five gene therapy drugs go hand in hand, which also poses a problem for the insurance system - how to deal with these many sky-high disposable drugs
with long-term benefits.
How the market reacts, how the insurance system handles, and how to form a commercialization model belonging to gene therapy will be answered
in 2023.
Figure 1 Gene therapy drugs to be commercialized in 2023 (Source: Official websites of each company; Organized by Fengshuo Venture Capital)
Bluebird's two products are in the spotlight, the company is on the verge of bankruptcy, and the commercialization of Skysona and Zynteglo drugs is the key to
survival.
However, the situation is not optimistic, the department is riddled with controversy over lentiviral therapy, and the two drugs were previously approved in Europe, but ended in commercial failure, it is reported that the price with European medical insurance parties has not been negotiated
.
On the other hand, the product of the rival CRISPR is about to catch up, and it is currently preparing to submit it for rolling review for listing
.
It is worth paying attention
to how Bluebird communicates and cooperates with US commercial insurance and medical insurance, and what sales strategy it can occupy enough markets before competing products go on the market.
For existing treatments, which are not fatal diseases, the market prospects of gene therapy drugs depend on the commercialization of Roctavian and Hemgenix
.
Roctavian and Hemgenix respectively treat hemophilia A/B, which are the first gene therapies
in their indications.
But hemophilia A / B has palliative care methods, the disease is not rapidly progressing, fatal, such diseases, gene therapy drugs advantage in ultra-long-term effect and reduce disability rate, improve quality of life, there are many similar diseases, we can take a peek at the sales of these two drugs
.
In addition, for untreated, fatal but very rare diseases, Upstaza will also hand over its commercial results
.
According to RBC analyst Brian
Abrahams said PTC has identified 300 patients, and he predicts PTC's annual revenue in Europe is $60 million
.
Upstaza is the first gene therapy
injected directly into the brain.
to inject directly into the brain for a fatal but very rare disease with no treatment.
2.
Listing approval node
Next year, more gene therapies may be approved, and different technologies will fully bloom, and successful approval will bring more certainty to gene therapy
.
.
Figure 2 Gene therapy drugs that may be marketed in 2023 (Source: official websites of various companies; Organized by Fengshuo Venture Capital)
Krystal
Biotech gene therapy B-VEC treats DEB patients
.
The first use of HSV vector, and gel, topical skin administration, phase 3 trial reached clinical endpoint, is expected to be successfully approved
.
If approved, B-VEC will also be the first gene therapy
for DEB patients.
In AAV, gene editing, lentiviral technology hot technologies are popular, Krystal
Biotech has built a market-unique HSV platform to differentiate itself
.
Its HSV carrier platform has been extended to multiple sites of administration, pointing to larger indications
such as cystic fibrosis.
BioMarin submitted the BLA to the FDA again on Roctavian, supplementing the longer-term data, and the two-year data showed the end point, and the FDA announced that it would no longer conduct an advisory committee meeting review and was expected to be approved for the US market
.
Sarepta gene therapy SRP-9001 for the treatment of DMD, which has shown positive therapeutic effects and consistent safety data in 1, 2 and 4 years of treatment, is expected to be approved
in May next year.
At the same time, Roche has acquired its commercial rights
outside the United States.
If approved, SRP-9001 would be the first gene therapy
for DMD patients.
Bluebird's third gene therapy drug, lovo-cel, is for the treatment of sickle cell disease, and the company expects to complete lovo-cel in the fourth quarter of 2022
Vector and drug product analysis of BLA is comparable and is expected to submit lovo-cel's BLA
in the first quarter of 2023.
While some of Lovo-Cel's clinical shelving is on hold, the company continues to communicate
with the FDA.
CRISPR's exa-cel treats transfusion-dependent β-thalassaemia and severe sickle cell disease
.
Exa-cel is moving fast, Vertex
/CRISPR
Therapeutics began filing a rolling listing application for exa-cel's BLA in November 2022 and expects to complete the submission
in Q1 2023.
In addition, Vertex has completed discussions with the EMA and MHRA on the data required to support the exa-cel listing application and is expected to submit the listing application
in the EU by the end of 2022.
If approved, Exa-Cel would be the first gene-editing therapy
.
Adenona's EB-101 treatment for DEB, which announced positive results from a Phase 3 trial in November this year, significantly improved healing and pain in large chronic RDEB wounds, met the primary endpoint, and is expected to submit a BLA application
to the FDA in Q2 2023.
Drugs that will be marketed next year and submitted to the FDA for review, HSV, CRISPR editing and other new technology drugs are applied for for the first time, and the FDA's review will also affect other companies in the technology field, and if successfully approved, it is a great benefit
to technology platform companies.
3FIC-gene-editing gene editing is perhaps the most anticipated field of gene therapy, and many of the popular FIC drugs were born here
.
Further comb through the current pipeline of gene editing companies to see how
these FIC drugs will fare next year.
Figure 3 Pipeline progress of gene editing companies (Source: official websites of each company; Organized by Fengshuo Venture Capital)
In 2023, the first product of the leading CRISPR company will submit a rolling listing application; VCTX210/211, a synonymous β cell replacement therapy for the treatment of diabetes, by CRISPR and ViaCyte, has entered clinical trials
.
Intellia, dedicated to in vivo gene editing, will launch a Phase II trial of NTLA-2002 for the treatment of hereditary angioedema in 2023H1, the first in vivo editing product; At the same time, its NTLA-2001 also entered clinical phase
I.
Base editing techniques are mixed
.
Beam's first pipeline Beam-101 for the treatment of β-thalassemia and sickle cell disease entered the clinic this year, which is the first base editing product
to enter the clinic.
On the other hand, Verve's product Verve-101 IND application has been put on hold, and previously, Beam's multi-base editing CAR-T therapy Beam-201 IND application was also put on hold by the FDA, requiring more safety data
such as off-target and rearrangement.
The above FIC drugs, which are based on the concept of gene editing, are often accompanied by huge uncertainty, but it is this uncertainty that makes every time a definite and positive development is announced, it provokes a huge chemical reaction
.
In 2023, expect more progress to be announced
.
every time a definite and positive development is announced.
4.
The initial period of domestic gene therapy: This year, the number of financing events in the domestic gene therapy field reached a record high, and the number of clinical trials approved reached a record high
.
As of the end of November, about 12 domestic gene therapy drug clinical trial applications have been approved, and a large number of clinical trials will be carried out next year, see the article previously pushed by the domestic gene therapy companies for details
In addition to the new drugs applied for clinical trials this year, there are some more advanced gene therapy drugs that may have more clinical data
next year.
Newforth's first product for the treatment of Leber hereditary optic neuropathy caused by ND4 mutations has entered clinical phase
3 this year.
Belief Pharma's first product for the treatment of hemophilia B entered the clinical trial stage in 2021.
This year, the drug was included in the breakthrough therapy variety by CDE, was recognized as an orphan drug by the FDA, and the IIT trial results were officially published in The
Lancet-Hematology.
Boyaji entered clinical trials in 2021 for the first product to treat transfusion-dependent β thalassemia
.
epilogue
After years of exploration, the foreign gene therapy market has gradually opened up, and a number of gene therapy drugs have been approved and more drugs are waiting to be approved, and there has been a trend
of "mass production".
If the listing and commercialization of this batch of gene therapy drugs is smooth, it will attract more funds and manpower, forming a perfect cycle
.
Domestic gene therapy companies have just started, and next year is a key starting period, and it is expected that the harvest of green fruits will not be until the year after that
.
Seize these key nodes and surprise you
.
