Broader use of an mRNA vaccine booster is still on the table in the U.S. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said that in addition to voting Oct. 14 on Moderna Inc.’s proposed 50-mcg COVID-19 booster to be used in people ages 65 and older and certain other at-risk groups, the Vaccines and Related Biological Products Advisory Committee (VRBPAC) would be asked to discuss whether updated information on the effectiveness of the vaccines supports use of both the Moderna and Pfizer Inc.-Biontech SE boosters in the general adult population. The committee had rejected that general use last month when it met to vote on the Pfizer booster.

Rectify raises a $100M series A to restore ABC transporters

Rectify Pharmaceuticals Inc. closed on a $100 million series A financing to fund development of a pipeline of therapies to restore ABC transporter function for treating genetic diseases. The family of 48 transporters are membrane-bound proteins with etiologic loss-of-function mutations that can be found in the lungs, liver, gastrointestinal tract, the eyes and the CNS. Jonathan Moore, Rectify’s chief scientific officer, founded the Cambridge, Mass.-based company in 2020. He is also a founding scientist at Vertex Pharmaceuticals Inc., where he developed cystic fibrosis therapies that target CFTR, one of the ABC transporter family members. The financing was co-led by Atlas Venture, Omega Funds, Forbion and Longwood Fund.

Tentarix closes $50M series A round to probe ‘combinatorial space’ for multifunctional drugs

Tentarix Biotherapeutics LP broke cover with $50 million in series A funding to take forward an industrialized flow cytometry platform that enables it to screen rapidly for multifunctional antibody-based drugs. The San Diego-based company has already demonstrated the feasibility of its approach by recapitulating the activity of interleukin-2 (IL-2) with a construct that targets two IL-2 receptor subunits, the beta and gamma chains.

Abalos adds $38M in series A extension to take virotherapy into the clinic

DUBLIN – Abalos Therapeutics GmbH raised €32.5 million (US$37.6 million) in a series A extension, taking the total raise to €43 million, enough to enable the company to generate clinical proof-of-concept data with its lead viral immunotherapy for cancer. The extension dwarfs the company’s initial raise, which it completed two years ago. “Initially, it was not the plan to go out and pitch again,” CEO Marcus Kostka told BioWorld. The original intention was to complete an internal round with its existing investors, but the Dusseldorf, Germany-based firm received some inbound interest at the same time, so it decided to gauge the level of market appetite for the company’s distinctive approach to immuno-oncology.

PTC vows to learn from Bluebird’s pricing woes as EU gene therapy decision approaches

PTC Therapeutics Inc. is already established as a player in rare diseases, working with Roche Holding AG to develop and market Evrysdi (risdiplam) to treat certain patients with spinal muscular atrophy. With Evrysdi now approved in the U.S. and Europe, and Translarna (ataluren) approved in Europe for Duchenne muscular dystrophy, South Plainfield, N.J.-based PTC is approaching a crucial juncture with its first gene therapy product. Moving into gene therapy was an obvious move for PTC after those first successes, but how will it overcome the market access challenges associated with those ultra-pricey treatments?

WEE1 researchers lining up to explore new cancer approach

Recent news that The University of Texas MD Anderson Cancer Center is teaming up with Schrödinger Inc. to work on the latter’s WEE1 inhibitor – along with data disclosed at the 2021 meeting of the European Society for Medical Oncology – shone a light on the gatekeeper checkpoint kinase, which a number of players are busy exploring, though nothing the class has been approved so far. Companies busy in the space include Astrazeneca plc, Debiopharm SA, Impact Therapeutics Inc., Nuvation Bio Inc. and Zentalis Pharmaceuticals Inc.

BIA Autumn Forum: Keep collaboration, innovation momentum post-pandemic, new UK science minister urges

LONDON – After the hard, solitary slog through months of pandemic, U.K. biotech finally convened in person, buoyed by the contribution the sector has made to dealing with COVID-19, and evidently pleased to be face-to-face once more. Moving on from the crisis, the industry is now poised for what was described as a “new era of biotechnology” that will build on successes such as the rapid development and manufacturing scale-up of COVID-19 vaccines, the setup of large-scale national clinical trials with innovative designs to test potential COVID-19 therapies, and taking an international lead in using genome sequencing to track the evolution of the SARS-CoV-2 virus.

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