Novartis to Acquire Kate Therapeutics for Up to $1.1 Billion to Strengthen Gene Therapy Pipeline
Kate Therapeutics
Gene Therapy Developer
Novartis
Drug Development and Manufacturing
Welcome to the 2024 Annual Meeting of the Hepatology New Drug Alliance and the Weight Loss New Drug Forum!
On November 21, 2024, local time, Novartis issued a press release announcing the acquisition of Kate Therapeutics for up to $1.1 billion.（Kate）。
Kate Therapeutics, Inc. focuses on developing adeno-associated viruses for the treatment of hereditary muscular degenerative diseases and heart diseases.（AAV）Gene therapy, currently its R&D pipeline indications cover X-linked myotubular myopathy.（XMTM）, Duchenne Muscular Dystrophy（DMD）Facioscapulohumeral Muscular Dystrophy（FSHD）, Type 1 Myotonic Dystrophy（DM1）And cardiomyopathy, etc. The above pipelines are still in the preclinical stage for now, among which the XMTM project is co-developed by Kate Therapeutics and Astellas. In addition to the existing pipelines, another significant asset of Kate Therapeutics is its established Deliver and Cargo technology platforms. Among them, Deliver is designed to screen evolutionary functional AAV capsids for more efficient and targeted delivery. Cargo, on the other hand, can perform large-scale parallel reporter gene analysis in vivo.（MPRA）By leveraging machine learning, develop stronger combinations of enhancers and promoters to facilitate the expression of target genes in specific cell types.
Novartis stated in its press release that the acquisition of Kate Therapeutics is mainly to complement its pipeline layout in the fields of gene therapy and hereditary muscular degenerative diseases.
The Original Intention Behind the Establishment of a Biotech Company
In June 2023, Kate Therapeutics announced the completion of a $51 million Series A financing, marking its emergence from stealth mode with its first public appearance. The company has four co-founders: Dr. Kevin Forrest, Dr. Sharif Tabebordbar, Dr. Mark Fielde, and Dr. Alan Beggs.
While researching the background of this fortunate biotech, I happened upon a New York Times interview with one of its founders—Dr. Sharif Tabebordbar. The article documented Dr. Tabebordbar’s career history and his original motivation for establishing this biotech, offering a perspective from the viewpoint of a patient’s family member, or even a carrier of a disease-causing gene, showcasing the suffering endured by those with hereditary muscular degenerative diseases. I have translated and organized this interview for your reference.
In 1986, Dr. Tabebordbar was born, and at that time, his father Jafar was 32 years old. Generally speaking, 32 is the prime of one’s life, but Jafar's body differed from others of the same age as he had already started experiencing discomfort caused by muscle atrophy, such as swaying left and right while walking and frequently tripping over. These symptoms worsened over time, and in his later years, Jafar could no longer drive a vehicle normally; by his 50s, he could only use one hand.
As far back as Dr. Tabebordbar can remember, he witnessed his father’s declining health. He wanted to know what the disease was and whether he and his younger brother, who inherited their father's DNA, might one day end up like him, confined to a wheelchair. But at that time, the scientific community had only just begun to recognize this rare disease, and no one could answer his question.
The strong desire to save his father took root in Dr. Tabebordbar's heart from an early age. During his later academic journey, Dr. Tabebordbar firmly chose the direction of biology and was successfully admitted to the accelerated program at Tehran University in Iran. It is worth mentioning that the program only admits 8-10 people each year, making acceptance a significant achievement.
After obtaining his master's degree from the University of Tehran, Dr. Tabebordbar successfully joined Professor Wagers' lab at Harvard University, fulfilling his wish to begin researching muscular dystrophy. Later, taking advantage of his father’s visit to Harvard for his graduation ceremony, Dr. Tabebordbar conducted genetic sequencing for his father but did not find the disease-causing mutation. After more complex testing, it was eventually determined that what had troubled his father for so many years was FSHD. Fortunately, although Dr. Tabebordbar inherited this mutation, the disease-causing gene was incomplete, so he did not develop FSHD.
FSHD is one of the typical rare diseases, with an incidence rate of approximately 1 in 20,000, making it the third most common neuromuscular disorder after DMD and DM. It is estimated that more than 870,000 people worldwide are affected by this disease. FSHD is autosomal dominant, but about 20% to 30% of patients have de novo mutations without a family history. Based on molecular genetic mutation characteristics, FSHD can be divided into: Type 1 FSHD (more common, accounting for about 95%) and Type 2 FSHD. FSHD mainly affects the facial, shoulder, and upper arm muscles of patients. In the later stages of the disease, about 20% of FSHD patients eventually require the use of wheelchairs. Currently, there are no drugs available on the market for treating or slowing the progression of FSHD.
After graduating from Harvard, Dr. Tabebordbar started his own business and founded a gene-editing therapy company, but unfortunately, it failed. After the failure of his first venture, Dr. Tabebordbar took a job at a research institute, focusing on screening millions of viruses to identify those that almost exclusively target muscles. This undoubtedly laid the foundation for his later co-founding of Kate Therapeutics, Inc. with several other founders.
"I conducted 100 experiments, but 95 of them did not succeed," Dr. Tabebordbar said in an interview. However, this is precisely the spirit that scientists need. Dr. Tabebordbar hopes to help more people like her father who have suffered from diseases to be free from pain. When talking about her father's life being tormented by illness, Dr. Tabebordbar expressed: "He was born at the wrong time." Her words were filled with regret.
Summary
Image Source: Novartis Official WebsiteReferences:
1.Official Websites of Various Companies
Seize the Opportunities of the Times, Overcome Stubborn Liver Diseases! — 2024 Liver Disease New Drug Alliance Annual Conference and Weight Loss New Drug Forum (First Round Announcement)
2024-11-20
2024-11-19
Copyright Statement/Disclaimer
This article is an original piece.
This article is for information exchange purposes only and does not provide any commercial, medical, or investment advice.
The images, videos, fonts, music, and other materials in the article are either authorized genuine works purchased by PharmaTimes, sourced from the WeChat public image library, taken from the company's official website or the internet. Some materials are used under the CC0 protocol, and their copyrights belong to the respective owners. PharmaTimes makes every effort to credit the sources.
If you have any questions, please contact us.
Sincerely grateful!
DrugTimes Official Website: www.drugtimes.cn
Contact Information:
Phone: 13651980212
WeChat: 27674131
Email: contact@drugtimes.cn
Register to attend and discuss the development of new drugs for liver disease and weight loss!