Gene Therapies

Deal Snapshot: The companies had announced the deal back in February 2021, but citing its “strategic focus,” AbbVie told Caribou it was backing out.

The company presented its pipeline to investors during an R&D day, outlining expansion plans for Voxzogo and Roctavian as well as several early pipeline drugs.

Otsuka steps into the ocular gene therapy field through a technology licensing deal worth up to $1.5bn with ShapeTX, which in turn further expands the US firm's global reach following a $3bn collaboration with Roche. 

With its first US study for a base-edited CAR-T therapy now back on track, Beam Therapeutics is gearing up to begin studies of its first in vivo candidate, BEAM-302, next year in AAT.

Among the latest financing deals, advanced therapeutics such as cell and gene therapies (CGT) continued enjoying the spotlight from venture capital and private equity investors in China. Meanwhile, small molecules with novel mechanism of action such as molecule glue stood out from their peers to win investors’ favor.

The French non-profit, whose IP is incorporated in Zolgensma, is taking its Crigler-Najjar gene therapy into a pivotal study on its own for now. It hopes to be ready for an EU filing in two years.

In this week's podcast edition of Five Must-Know Things: a look at global executive compensation; Pfizer’s new myeloma approval; Merck & Co. and Kelun trump ADC breast cancer success; combo Alzheimer’s therapies in Eisai’s pipeline; and a look at the state of gene therapy.

Precision Biosciences is exiting the allogeneic CAR-T field after four years in early clinical trials, selling off its former lead asset for just $21m.

The US biotech’s TSHA-102 has shown surprisingly early signs of functional improvement in a single patient treated in its Phase I/II Rett syndrome trial, promoting a major stock surge supported by the news of a $150 private placement and regulatory updates.

BioMarin, Sarepta and bluebird bio are all launching new gene therapies in the US. The drug makers updated investors on the commercial execution during second quarter financial earnings calls.

The US FDA could approve as many new gene therapies in 2023 as it has in the previous five years. Development in the field has rapidly accelerated, but still faces hurdles.

The US biotech revealed the US Food and Drug Administration would be holding an advisory committee meeting for exa-cel during its second quarter earnings, but analysts are unfazed by the news amid robust revenues and raised guidance.

Private Company Edition: Kriya raised the first $270m of its now $430m-plus series C venture capital round in May 2022 and has raised more than $600m to date. Also, Nexo launched with $60m in series A cash, Ossium brought in $52m in series A financing and cell therapy CDMO Kincell launched with $36m.

The drug-pricing watchdog gave the gene therapy for metachromatic leukodystrophy an “A” grade for use in presymptomatic patients receiving the therapy, which already is approved in Europe.

South Korea's SK Biopharmaceuticals unveils a new long-term road map, or "financial story", which involves generating substantial cash flow and expansion into new modalities with the ultimate goal of becoming a “balanced, big biotech.”

Biopharma fears that the Joint Clinical Assessment, a first step to harmonizing health technology assessments in the EU, is not fit for purpose for cell and gene therapies and will increase rather than eliminate market access delays.