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Week of October 26

This weekly post looks to identify important advancements in the areas of oncology, CNS disorders and neurodegeneration, technologies, and industry and corporate developments. More in-depth reading and analysis can be found in the attached links.


  • Turning Point Therapeutic’s Phase 1 study of TPX-0022, a MET inhibitor, demonstrated clinical efficacy and safety across multiple tumor types. 9 out of 15 evaluated advanced solid tumor patients with MET-dysregulation achieved clinical benefit, confirmed or unconfirmed partial response or stable disease. Link

  • Forma Therapeutics announced positive interim Phase 2 clinical results for olutasidenib to treat IDH1m relapsed/refractory acute myeloid leukemia (AML). While Olutasidenib achieved their primary endpoint of a composite complete remission (CR+CRh) rate of 33.3%, olutasidenib did not meet their CR+CRh median duration. Link

CNS Disorders and Neurodegeneration

  • Sanofi and the Parkinson's Foundation announced a collaboration in the US to advance the availability of genetic testing and counseling for Parkinson's disease patients. Over the next 2 years, Sanofi will fund the PD GENEration initiative which test patients for key Parkinson's-related genes, GBA, LRRK2, and SNCA, and identify if they qualify for certain clinical trials. Link


  • CRISPR Therapeutics’ CTX110 utilizes CRISPR/Cas9 to develop an allogeneic CD19 CAR T-cell therapy from healthy donor samples to treat relapsed or refractory CD19+ B-cell malignancies. Early Phase 1 results found that 4 out of 11 patients achieved a CR. However, one patient who received the highest dose died, leading to a pause in enrollment for the high dose arm of the trial. Link

  • Intellia Therapeutics received authorization from the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to start their Phase 1 trial evaluating NTLA-2001 treatment of patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). NTLA-2001 also utilizes CRISPR/Cas9 to knockout and reduce the levels of mutant TTR. Link

Industry and Corporate Developments

  • Bayer agreed to acquire Asklepios Biopharmaceuticals (AskBio) for $2B allowing Bayer to become a player in gene therapy development. AskBio has several pre-clinical and clinical AAV-based gene therapy candidates to treat Pompe disease, Parkinson’s disease, and congestive heart failure. Link

  • Be Biopharma, a biotech company looking to develop cell therapies from B-cells, raised $52M in Series A funding from Atlas Venture and RA Capital Management. Funds will be used to develop B-cell therapies to treat a variety of diseases. Link

  • Sarepta Therapeutics raised $107M for its new startup, AavantiBio, which will focus on developing gene therapies for rare genetic diseases. Link

  • Ultragenyx and Solid Biosciences announced a strategic collaboration to develop and commercialize new gene therapies for Duchenne Muscular Dystrophy (DMD). Solid Biosciences received $40M upfront with $255M in cumulative milestone payments for an exclusive license for their AAV8 gene therapy to treat DMD and other diseases. Link

  • Roche announced a collaboration with Atea Pharmaceuticals worth $350M to jointly develop Atea’s orally administered direct-acting antiviral to treat COVID-19 outside the hospital. Link

  • Pfizer’s Hospital Business agreed to acquire Arixa Pharmaceuticals. The main driver of this deal is Arixa’s lead candidate, ARX-1796, an oral prodrug of a beta-lactamase inhibitor to treat Gram-negative bacterial infections. Link

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