With the number of high-priced cell and gene therapies expected to grow dramatically over the next decade, U.S. Sen. Bill Cassidy (R-La.) is asking for input from patients, doctors, manufacturers, payers and policy wonks on how to pay for the treatments and ensure patient access when they become more mainstream across the ultra-rare disease spectrum.
In one of the larger biopharma IPOs in 2023, Cargo Therapeutics Inc. pulled in $281.3 million on Nov. 10, selling 18.75 million shares at $15 each, the low end of its price range. The market debut comes just eight months after the San Mateo, Calif.-based company raised $200 million in an oversubscribed series A round.
Astrazeneca plc is making a $220 million equity investment and tossing in $25 million up front to Cellectis SA as part of a new collaboration agreement. The deal is part of Astrazeneca’s efforts, including a July licensing agreement worth about $1 billion with Pfizer Inc., to delve deeper into gene therapy for treating cancer and rare diseases.
Moderna Inc. has more than COVID-19 vaccines in the hopper, and the company aims to add still more oomph by way of an oncology deal with Immatics NV that could be worth more than $1.7 billion for the latter, which banks $120 million up front and stands to collect research funding as well.
Tolerogenixx GmbH raised €7 million (US$7.6 million) in an extension to its series A round, which will enable it to continue phase IIb development of a cell therapy that induces donor-specific immune tolerance in kidney transplant recipients. At the same time, the company disclosed five-year follow-up data from a phase Ib trial of the therapy, which demonstrated that recipients continue to have stable graft function and to avoid acute rejection and severe opportunistic infections while on a reduced regimen of immunosuppressive drugs.
Lion TCR Pte. Ltd. raised $40 million in series B funding that will see the company advance its clinical trial program for its hepatitis B virus (HBV)-specific T-cell receptor (TCR) cell therapy for HBV-related liver cancer.
Two years after a U.S. FDA advisory committee agreed that Celltrans Inc.’s pancreatic islet cellular therapy had a favorable risk-benefit profile, the agency has finally approved Lantidra (donislecel) for treating adults with type 1 diabetes.
Regulatory T-cell specialist Quell Therapeutics Ltd. has sealed a potential $2 billion agreement under which Astrazeneca plc is taking rights to two autologous Treg cell therapies for treating inflammatory bowel disease (IBD) and type 1 diabetes. Quell will receive $85 million up front, the majority of which is in cash, with a modest (undisclosed) equity investment. Reaching the $2 billion headline figure will involve a series of development and commercial milestones and royalties on sales.
For Jeff Galvin, the CEO and founder of newly launched Addimmune Inc., HIV is not a condition that’s in the rearview mirror. It needs a functional cure to save lives, make people healthier and save money that need not have been spent. People wonder why it’s worth bothering to cure HIV, Galvin told BioWorld, when they are taking their medications every day and they are feeling pretty close to normal. But it’s not close for Galvin, who noted that there are side effects from taking the pills that can cause headaches, fatigue, nausea and diarrhea.
In its bid to become, as Chairman and CEO David Hallal said, the “world’s most indispensable cell and gene therapy technology company,” Elevatebio LLC disclosed a $401 million series D round with support from new and existing investors. At the same time, the company’s Life Edit Therapeutics Inc. affiliate inked a potential billion-dollar collaboration focused on gene editing therapies.