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Aug 26, 2021
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Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.
Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks. The company will utilize this technology with Roche in preclinical studies against targets pertinent for these neurological conditions and certain rare diseases.
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The collaboration may also utilize the biotech company’s technologies for gene delivery. This system is designed to deliver RNA editing technology or other payloads directly to particular body areas, such as the nervous system or muscle.
Shape focuses on unlocking the next breakthrough in RNA technologies in the gene therapy space across many therapeutic areas, as informed by Francois Vigneault, co-founder, and CEO.
Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases. The company’s gene delivery technology is based on AAV vectors, a platform lately utilized for various rare conditions treatment.
Under the agreement, Shape is eligible to receive an initial payment and payments for hitting development, regulatory and sales milestones potentially exceeding USD 3 billion in aggregate value. Any products from the collaboration will be developed and commercialized by Roche.
AllStripes has secured USD 50 million to accentuate its efforts at garnering medical records and finding them for insights, which could aid in drug development.
Formerly known as RDMD, AllStripes utilizes its dedicated platform to garner real-world data and medical histories from people with rare diseases such as Batten disease, Hunter syndrome, sickle cell anemia, and more. The company then utilizes the de-identified records to improve the design of clinical trials and quality of life measurements.
Its series Previous investor Lux Capital led b round with additional backing from Jazz Venture Partners, Spark Capital, Medidata Solutions, McKesson Ventures, and Maveron, plus angel investors comprising Komodo Health CEO Arif Nathoo, M.D., and Leila Zegna, director of the Kabuki Syndrome Foundation.
Currently, AllStripes partners with more than 30 patient advocacy organizations to back research into 40 different conditions by gathering data from more than 3,000 users to date.
The company has also worked with drug developers such as Novartis, HemoShear Therapeutics, Taysha Gene Therapies, UCB Biopharma, and the University of Pennsylvania.
Real Chemistry and Datavant know how to combine forces and synthesize data for big decisions. Real Chemistry gobbled up 12 companies, hired 600 people in 2020, and rebranded in March. Meanwhile, in June, Datavant inked a USD 7 billion deal to combine with medical records platform Ciox Health.
Now, Real Chemistry and Datavant are getting real with a plethora of data that the pair contemplates can help deliver on new gene therapies and precision medicines. The companies are linking arms, for an unrevealed amount, to support biotech and pharmaceutical companies connecting their proprietary de-identified first-party data with real-world data.
Through its machine-learning unit IPM.ai, Real Chemistry has more than 300 million de-identified patient journeys and an eye-popping 65 billion anonymized social determinants of health signals as it is ready. IPM.ai was swooped up by Real Chemistry in January.
That seemingly never-ending treasure chest of data could be paramount in diagnosing the patients eligible for gene therapies and precision medicines. Because of difficulty diagnosing those patients, the population is underserved, as per said by the companies.
Through the collaboration, the companies focus on connecting clinical trial data with specialty pharmacy information, natural history, registry data, and broad claims databases. Bringing all of that information together will ideally reduce the diagnosis window, lead to more personalized therapies and enhance clinical trials while protecting patients’ privacy, as told by Jonathan Woodring, IPM.ai’s general manager, in a statement.
The U.S. government has continued to eye on new, better anthrax vaccines despite the availability of Emergent BioSolutions’ Biothrax. Now, BlueWillow Biologics, working with Porton Biopharma, has delivered phase 1 data on a nasal vaccine, which it claims to have more advantages over other options.
The trial randomized 84 healthy volunteers to administer one of two doses of BW-1010 via a sprayer or pipette, a placebo, or active control. The study looks at the frequency of solicited systemic and local reactions in the 39 days after dosing, severe events out as far as Day 393, and changes in the levels of neutralizing antibodies at Day 57 and Day 85. Researchers will compare the two-time points to recognize the optimum timing of peak immune response following nasal vaccination.
BlueWillow shared little data about how the vaccine performed in its interim analysis. Giving two intranasal doses four weeks apart were found to be safe and well-tolerated; BlueWillow apprised and induced systemic and mucosal immunity as shown by levels of neutralizing systemic antibodies, nasal IgA/IgG, and Th17 response in peripheral blood cells.
The study is set to conclude in the fall. BlueWillow intends to take the anthrax vaccine through further clinical trials after looking at phase 1 data, positioning it to find out whether BW-1010 has a future in a space targeted by multiple other companies.
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