Gene therapy biotech Kriya Therapeutics has acquired Tramontane Therapeutics, a startup focused on metabolic diseases. Tramontane brings a gene therapy that’s a potential one-time treatment for the fatty liver disease NASH.

The FDA approved Roctavian for treating hemophilia A. The regulatory decision makes the BioMarin Pharmaceutical product the first gene therapy for this inherited bleeding disorder.

Sarepta Therapeutics’ Elevidys is now the first FDA-approved gene therapy for Duchenne muscular dystrophy. Elevidys is an engineered version of a gene intended to restore function lost to the mutation at the root of the inherited muscle disease.

Multiple efforts are underway to improve cell therapies for cancer. During the MedCity News INVEST conference in Chicago, a panel discussed the future of cell therapies and what the industry doing to manage the financial risks of these new treatments.

Companies presenting their technology at the conference seek to support a diverse patient population spanning conditions such as inflammatory bowel disease, food allergies, and cancer. Cell, gene therapy and immunotherapies also form some of their treatment approaches in development.

By selling partial royalties to approved hemophilia gene therapy Hemgenix, uniQure gains immediate cash to support its pipeline, including a Huntington’s disease gene therapy already in the clinic . The royalty deal extends uniQure’s cash runway into 2026.

A panel discussion at the annual Abarca Forward conference in Puerto Rico last month sought to identify some of the innovative financial approaches that can be applied to managing the high price of novel therapies. Conversations also addressed what payers need for these financing models to be sustainable.

VintaBio manufactures AAV and lentiviral vectors for entities pursuing clinical development of cell and gene therapies. The contract development manufacturing organization’s launch comes as demand for viral vectors continues to outpace the supply of these crucial components of the therapies.

BioMarin Pharmaceutical’s submission of additional data for patients treated with its hemophilia A gene therapy, Roctavian, mean that the FDA will push out a regulatory decision to early summer. But it could be worth the wait as analysts anticipate FDA approval and project Roctavian becoming one of BioMarin’s biggest products.

AbbVie and Capsida Therapeutics are expanding their gene therapy R&D alliance to the eyes. Capsida is in line to receive $70 million now and up to $595 million later, depending on the progress of the eye programs.

Panelists at a recent pharmacy conference were optimistic about the future of value-based drug contracting. For example, one said that healthcare will be close to establishing a value-based formulary by 2030, and another said that longitudinal patient tracking will become a more important part the drug pricing process.

Taysha Gene Therapies has encouraging data from an open-label, Phase 1/2 study in the rare disease giant axonal neuropathy. It might not be enough. The FDA recommended the biotech conduct a randomized and placebo-controlled study—a challenge in any ultra-rare disease.

Vector BioMed has developed a technology platform for designing and manufacturing the lentiviruses used to deliver cell and gene therapies. The startup is launching as the field continues to experience shortages of these viral vectors.

Astellas has become an active player in gene therapy. MedCity News caught up with company executive Richard Wilson, who explained Astellas’s strategy, the clinical holds on its two most advanced programs, and the rationale behind its recent gene therapy deals.

Neurocrine Biosciences is paying $175 million up front for rights to a preclinical Voyager Therapeutics gene therapy that could put the biotech in competition with Eli Lilly among other companies. Voyager could earn up to $4.2 billion in milestone payments.

Fewer new drugs won FDA approvals in 2022 compared to previous years, but some of the products that passed regulatory muster are indicative of trends expected to continue into 2023. For example, more gene and cell therapies are on track to face regulatory decisions in the coming year.

Eli Lilly is paying $55 million up front to begin an alliance with Capsida Biotherapeutics pursuing gene therapies for central nervous system disorders. Capsida’s technology enables the delivery of a gene therapy to a single organ or multiple organs, a capability that could bring such treatments to a wider range of diseases.

A Ferring Pharmaceuticals gene therapy is now FDA approved for treating bladder cancer that does not respond to an immunotherapy used to treat the cancer in its early stages. The Ferring gene therapy, Adstiladrin, turns bladder wall cells into tiny factories churning out a cancer-fighting therapeutic protein.

2023 is shaping up to be a crucial year for everyone involved – and affected – by the pharmaceutical industry. It’s likely to be a year of experimentation for payers – and defensiveness for drug makers.

As gene sequencing, machine learning, powerful imaging, sensors, gene and cell therapies make their way out of academic labs into everyday healthcare, our approach to healthcare is changing. For the first time, not only can we pinpoint the molecular and genetic causes of disease, but we also have the technologies to access them. On the back of this, prevention and cures are becoming possible. Can we afford them?

CSL Behring’s hemophilia B gene therapy, Hemgenix, carries a $3.5 million list price. But the company contends that by reducing economic burdens associated with bleeding and prophylactic infusions, the one-time treatment will save money over time.

Telaria is developing a gene therapy for the rare skin disorder recessive dystrophic epidermolysis bullosa. The company was formed by Replay, a gene therapy biotech that forms subsidiaries that leverage its platform technologies for writing and delivering genetic medicines.

Cell and gene therapies are offering patients potentially curative treatments for a growing scope of diseases. Insurance companies are trying to figure out how to pay for them. Industry consultants speaking at the HLTH conference offered some strategies they see payers taking to these new therapies.

Astellas has purchased an equity stake in Taysha Gene Therapies in a $50 million deal that gives it an option to license the company’s two lead gene therapy candidates. The Japanese pharmaceutical giant also gains the option to buy its new partner outright depending on clinical and regulatory developments expected in the coming year.

Specialty carve outs that move specialty drugs away from traditional prescription drug management to a pharmacy benefit administrator (PBA) or specialty administrator will continue and likely to provide cost savings.

Gene therapy can offer hope to patients with previously untreatable diseases, but there’s more work to be done to improve both the safety and efficacy of this therapeutic modality.

Eli Lilly is acquiring Akouos on the heels of the biotech receiving FDA permission to begin a clinical trial testing its gene therapy for an inherited form of hearing loss. The deal is Lilly’s second gene therapy acquisition following the 2021 buyout of Prevail Therapeutics.

BioMarin Pharmaceutical said the layoffs will improve efficiency across the company as it prepares for the potential U.S. launch of Roctavian, a gene therapy for severe hemophilia A. The one-time treatment was approved in Europe in August.

AstraZeneca’s LogicBio acquisition comes at a critical time for the biotech. Though LogicBio has encouraging early clinical data for its lead gene-editing therapeutic candidate, the company’s cash woes include a flagging stock price that is set to lose its listing on the Nasdaq.

CEO and Co-founder Daniel Oliver share how he and his co-founder started the company and their vision for treating age-related conditions.