Cell therapy weekly: Takeda and Code Biotherapuetics enter strategic collaboration of potentially US$2 billion

Written by Sarah Rehman

This week: Takeda and Code Biotherapeutics enter strategic collaboration of potentially US$2 billion, Eli Lilly launches the Lilly Institute for Genetic Medicine with a US$700 million investment and Homology Medicines’ stocks fall as clinical hold is placed on phenylketonuria gene therapy candidate.

The news highlights:


Takeda and Code Biotherapeutics enter strategic collaboration of potentially US$2 billion

Code Biotherapeutics (PA, USA), a gene therapy company, has announced a strategic collaboration with Takeda (Tokyo, Japan), a patient-focused global pharmaceutical company. The deal of potentially US$2 billion will utilize Code Bio’s 3DNA® non-viral genetic medicine delivery platform to develop targeted gene therapy for a liver-directed disease program. The agreement will also permit additional study into central nervous system-directed rare disease programs. The two companies have confirmed that they will collaborate on research activities up to candidate selection, after option exercise, Takeda will take sole responsibility for further development and commercialization.

“This collaboration will further enhance our capabilities by bringing together Takeda’s expertise in gene therapy and rare diseases with our expertise in delivering genetic medicines as we drive forward on our mission to bring meaningful treatments and cures to patients suffering from serious and life-threatening genetic diseases,” commented Brian P. McVeigh, Code Bio Chairman, Chief Executive Officer and Co-Founder.

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Eli Lilly launches the Lilly Institute for Genetic Medicine with a US$700 million investment

Eli Lilly and Company (IN, USA), an American pharmaceutical company, has announced the launch of the Lilly Institute for Genetic Medicine, with a US$700 million investment to develop a research facility in the Boston Seaport area. The launch aims to foster collaboration between researchers in Boston and New York to leverage RNA and DNA-based technologies to produce therapies to treat and prevent disease. This will expand the company’s expertise in the genetic medicine space, which already accounts for more than 20% of their diabetes, immunology and central nervous system research portfolio. The facility is estimated to grow rapidly, with the number of scientists employed is expected to increase from 130 to over 250 within 5 years.

“Establishing the Lilly Institute for Genetic Medicine will allow us to pair cutting-edge technologies with our deep biological expertise in several areas including neuroscience and diabetes,” remarked Andrew Adams, Vice President of genetic medicine at Eli Lilly.

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Homology Medicines’ stocks fall as clinical hold is placed on phenylketonuria gene therapy candidate

Homology Medicines (MA, USA), a genetic medicines company, has announced that the FDA has placed a clinical hold on its pheNIX gene therapy trial of HMI-102 in adults with phenylketonuria, a rare but potentially serious genetic disorder. The clinical hold is prompted by observations in the study revealing elevated liver function tests, which consequently require modified risk mitigation measures. The announcement triggered a plunge in Homology’s shares, which were down 27% to US$2.82 on Tuesday.

“This hold on our PKU gene therapy trial is based on clinical observations in the pheNIX study and does not relate to CMC/manufacturing capabilities or Homology’s other clinical programs. We plan to provide next steps once we have more information following our FDA interactions,” stated Arthur Tzianabos, President and Chief Executive Officer of Homology Medicines.

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