The Problem of Gene Therapy Commercialization

Recently, a news, on August 17, US time, the FDA announced that Bluebird Biology's lentiGlobin-based lentiGlobin lentiglo gene therapy Zynteglo was announced for the treatment of patients with β thalassemia who need to receive routine red blood cell transfusions

Since the concept of "gene therapy" was proposed in the 1970s, the world has favored this sci-fi technology

Against this backdrop, Bluebird Bio landed on the NASDAQ in 2013 and was priced at $17 per share before listing, and the listed stock rose 50% to nearly $26 per share

In 2019, Bluebird Biologics' first gene therapy, Zynteglo, was approved for conditional marketing in the European Union, but the commercialization process in Europe was disastrous, and the first commercial treatment

The Problem of Gene Therapy Commercialization

Commercialization includes four aspects: market, pricing, production, and technology, but gene therapy does not seem to have an advantage

Gene therapy is often adapted to rare diseases, which are small in size, meaning that the market for antibodies and molecular drugs is not as broad

A classic example is the $1 million-priced Glybera, a drug with rare indications (used to treat lipoprotein lipase deficiency, a disease that has only about 1,200 patients in Europe), was approved for use by only one patient for 5 years, failed in commercialization, and ended in 2017

To this end, gene therapy companies have proposed a variety of payment schemes, including installment payment, extended payment, and payment according to curative effect

In terms of research and development and production, gene therapy requires higher

Dr.

High prices, fewer indications, complex technical mechanisms, high-threshold process development, large-scale production, strict regulatory requirements, and limited industrialization experience have all contributed to the commercialization of gene therapy as a difficult and dangerous road

When it comes to the most successful gene therapy drug, that is, Novartis Zolgensma, it may be possible to see the beginning

Zolgensma is currently approved in nearly 42 countries around the

SMA is the "number one genetic disease killer" among infants and young children under 2 years old, and it is not a rare disease, there will be one carrier in every fifty people, and once both husband and wife are carrying, the child has a 25% chance of getting sick

Before Zolgensma was listed, SMA also had a gene therapy drug, Spinraza (Spinraza, which has been included in medical insurance in China), and Nocinasine sodium needed to be injected annually
.
Zolgensma as a one-time therapy is about 20 times the price of nonsinasone sodium, and the emergence of Zolgensma has preempted many patients
who were once treated with nocinasone sodium.

In terms of payment, Zolgensma was included in Japan's medical insurance in 2020, and patients only need to pay 30% of the cost; It was incorporated into the National Health Service in the UK in March 2021
.
In the U.
S.
market, Novartis and related insurance companies work together to allow patient families to pay in installments of $5 years, averaging $
425,000 per year.
Due to the high degree of marketization of medical services in Europe and the United States, the penetration rate of commercial insurance among the public is relatively high
.
Among the people who buy insurance, the proportion of people who can afford high-priced drugs has increased on a large scale, and Zolgensma's success is also traceable
.

Notably, just a few weeks ago, on August 11, Novartis reported the deaths
of two children from acute liver failure after receiving Zolgensma gene therapy for spinal muscular atrophy.
When Zolgensma was first listed, there were industry questions about its unknown safety risks and long-term efficacy, which also made the health insurance department hesitate
.
The incident also confirmed the need to sound the alarm about the safety of gene therapy
.

Currently, Zolgensma's domestic (OAV101) clinical trial applications have obtained implied permission for clinical trials
.
According to public information, the drug was officially launched in Peking University First Hospital in June this year, and the clinical trial belongs to the Chinese part of the global phase III clinical STEEL study, which is aimed at the treatment of type 2 spinal muscular atrophy at the age of 2 to 18
.

summary

The current gene therapy drug production is similar to the monoclonal antibody drugs of the 1980s and 1990s, and the maturity of the production stage is 20-30 years
behind that of the monoclonal antibody drug.
There is no doubt that gene therapy has great promise in treating diseases, but there are also many challenges to be overcome
on the road to commercialization.
With the continuous development of the technology of various innovative companies and the emergence of CDMO that is constantly deeply rooted in gene therapy, there will be more optimized, lower cost and more efficient production methods in the future, the price of gene therapy will gradually decline to benefit more patients, and the treatment potential will continue to be released
.
At that time, gene therapy will be able to overcome today's problems and will become an indispensable presence
in biological medicine.

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baidu.
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zhihu.
com/p/430647774

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