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Dr. Gaurav Shah: Rocket’s Chief Gene Therapist & Virtuoso

Rocket Pharmaceuticals CEO Dr. Gaurav Shah shares the parallels between his leadership in the gene therapy space and his successes as a Grammy®-nominated recording artist with the Business of Biotech podcast.

endpoints

New targeted therapy approaches win Rain Therapeutics $63M — designed to beat a quick path to approval

When the biotech got started in the San Francisco Bay Area, it was singularly focused on tarloxotinib, a small molecule inhibitor named for its design to target low oxygen levels in the tumor and thereby sparing healthy tissues. More than two years later, Rain has tripled its pipeline within days, first licensing a research program from Drexel University, then more recently nabbing a Phase II-ready drug from Daiichi Sankyo.

Celyad’s High Hopes for a Path Forward in Cancer Immunotherapy with CYAD-211

Celyad Oncology is at the forefront of cutting-edge immunotherapy and is hopeful of providing a new way forward for patients with relapsed/refractory multiple myeloma. After receiving FDA approval on July 14th to begin Phase I trials, they plan to be in the clinic with their first patient the end of 2020.

global genes

X4 Pharmaceuticals’ Phase 2 WHIM Treatment Data Published in the Journal Blood

X4 Pharmaceuticals said that comprehensive data from its mid-stage trial of mavorixafor for the treatment of adult patients with the rare genetic syndrome WHIM was published in Blood, the official journal of the American Society of Hematology.

ShRNA and Single Genetic Modification May Lower CAR-T Manufacturing Costs

Cell therapy developer Celyad Oncology is using short-hairpin RNA (shRNA) to create T cells that can be used as an “off-the-shelf” allogeneic therapy for cancer patients. They are also using the technology to help T cells survive longer in patients.

contagion

Heat Biologics gp96 Vaccine Effort Aims for T Cell Immunity

Heat Biologics CEO Jeff Wolf talks with Contagion about the firm’s effort to develop a vaccine for the coronavirus focused on combining antibody and T cell immunity using the GP-96 protein.

Drug Targets Identified More Discerningly

A distinct targeting approach is being followed at Vaccinex. “We are not targeting the mutation that triggers the disease, but the pathological process that is triggered by the accumulation of misfolded proteins,” says Maurice Zauderer, PhD, the company’s CEO.

Companies To Watch: CalciMedica

CalciMedica has been developing a new class of anti-inflammatory drugs, calcium release-activated calcium (CRAC) channel blockers, with the lead indications for treating acute pancreatitis and viral pneumonia now in Phase 2 trials for its compound, Auxora, an injectable emulsion. Recently, the company discovered the same drug may have a large impact on systemic inflammatory response syndrome (SIRS), the hyperimmunity associated with viral pneumonia, a common comorbidity and comortality condition in COVID-19 disease. Hence, it now has a Phase 3 trial underway with Auxora targeting severe COVID-19 pneumonia.