cell therapy

Vertex Pharma’s Type 1 Diabetes Cell Therapy Shows Potential to Achieve Insulin Independence

In Phase 1/2 results for 12 patients at least one year after receiving the cell therapy, zimislecel, Vertex Pharmaceuticals reported all participants achieved blood glucose in the target range and 10 of the 12 were free of exogenous insulin as of the data cutoff. Vertex expects to complete enrollment in the pivotal portion of the study this summer, paving the way for potential regulatory submissions next year.

By Frank Vinluan

Pharma BioPharma,

Galapagos Says ‘No’ to SpinCo and Instead Will Seek Deals for All Assets

Galapagos has dropped plans for a business separation that would have enabled the legacy company to focus exclusively on developing new cancer cell therapies. Instead, Galapagos said it will pursue strategic alternatives for its assets.

By Frank Vinluan

BioPharma

FDA Approves Abeona Gene Therapy for Healing Wounds From Rare, Inherited Skin Disease

Abeona Therapeutics makes Zevaskyn by engineering a patient’s biopsied skin cells to express the collagen that they lack, then growing those cells into sheets that can be surgically applied to a wound. Abeona's cell-based gene therapy is just the third FDA-approved treatment for epidermolysis bullosa, a rare disease that leads to skin described as thin and fragile like a butterfly’s wings.

By Frank Vinluan

BioPharma

CRISPR Biotech Caribou Trims Pipeline and Cuts 32% of Staff to Focus on Two Cancer Cell Therapies

Caribou Biosciences’ restructuring narrows the biotech’s focus to two off-the-shelf cell therapies for blood cancers, whose key data readouts have been pushed out to the second half of 2025. It’s the CRISPR-editing company’s second cash-saving restructuring in the past year.

By Frank Vinluan

BioPharma, Pharma

Ouster of Peter Marks & Mass FDA Layoffs Have Biotech Sector Bracing for Regulatory Disruptions

As the FDA’s top biologics official, Peter Marks oversaw regulation of cell and gene therapies. The departure of Marks and mass layoffs throughout the FDA raise concerns about the agency’s ability to review and potentially approve such treatments.

By Frank Vinluan

BioPharma, Pharma

Vertex Cell Therapy Misses in the Clinic, But It Has Other Shots on the Type 1 Diabetes Goal

Vertex Pharmaceuticals is stopping development of VX-264, an experimental cell therapy/medical device treatment for type 1 diabetes, after an early look at Phase 1/2 data fell short of efficacy goals. This program came from a $950 million acquisition in 2019 that marked Vertex’s entry into type 1 diabetes.

By Frank Vinluan

BioPharma, Pharma

AstraZeneca Aims to Make Cell Therapy More Accessible With $425M EsoBiotec Acquisition

AstraZeneca’s EsoBiotec acquisition is the latest in a series of deals struck by the pharma giant to secure a place in the next wave of cell therapies for cancer and immune-mediated diseases. EsoBiotec’s allogeneic cell therapies are made by the in vivo reprogramming of a patient’s immune cells.

By Frank Vinluan

Pharma, BioPharma

Bristol Myers Squibb Buys Out Cancer Cell Therapy Partner 2seventy Bio in $286M Deal

Multiple myeloma cell therapy Abecma is the lone remaining asset of 2seventy Bio, which spun out from Bluebird Bio in 2021. 2seventy and Bristol Myers Squibb currently share in U.S. commercialization of Abecma.

By Frank Vinluan

BioPharma

Gene Therapy in an Implant: Neurotech Lands First FDA Approval in Rare Vision Disorder

Neurotech Pharmaceuticals’ surgically implanted cell and gene therapy, Encelto, is now FDA approved to treat macular telangectasia type 2 (MacTel). It’s the first therapy for this rare retinal degeneration disorder.

By Frank Vinluan

BioPharma

Safety Risk Sinks Cargo Therapeutics’ Cancer Cell Therapy Prospect, Leading to 50% Staff Cut

Phase 2 data for Cargo Therapeutics’ CD22-targeting cell therapy show low durability and cases of a severe immune response, some that were classified as grade 4 or 5 serious adverse events. Cargo is discontinuing the study and shifting focus to a different cell therapy on track to begin Phase 1 testing in mid-2025.

By Frank Vinluan

Pharma BioPharma,

Galapagos CEO Tells JPM Audience the Rationale for Planned Business Separation

Galapagos and Gilead Sciences have both evolved since inking their R&D collaboration agreement in 2019. Separating Galapagos into two offers a way for that company to pursue its cell therapy strategy while also keeping the Gilead alliance alive, executives from both companies said.

By Frank Vinluan

Pharma BioPharma,

Galapagos Keeps Focus on Cancer Cell Therapy, Spins Out Rest of Biz Into NewCo Backed by €2.4B

Galapagos said the planned business separation will leave the legacy company free to focus on developing cell therapies for cancer. An accompanying corporate restructuring will shave Galapagos’s headcount by 40%.

By Frank Vinluan

BioPharma, Pharma

ASH 2024 Recap: Movement in Multiple Myeloma, Cell Therapy, Sickle Cell Disease & More

A multiple myeloma cell therapy has encouraging results analysts say could make it best in its class and a gene-editing therapy is showing early promise in sickle cell disease. Here’s a recap of those developments and others from the annual meeting of the American Society of Clinical Oncology.

By Frank Vinluan

BioPharma, Pharma

Safety Stands Out in Arcellx Cell Therapy’s ASH Data, But Don’t Overlook Manufacturing Advantages

Arcellx’s cell therapy for multiple myeloma has shown no signs of the parkinsonism complication observed in clinical testing of Carvykti, a rival product from partners Johnson & Johnon and Legend Biotech. Arcellx is developing its CAR T-treatment under a partnership with Gilead Sciences.

By Frank Vinluan

BioPharma, Pharma

Roche Expands Its Scope in Allogeneic Cell Therapy With $1B Poseida Therapeutics Acquisition

Roche is acquiring Poseida Therapeutics, a biotech developing allogeneic cell therapies for cancers and immunological indications. The two companies have been partners in hematological malignancies since 2022.

By Frank Vinluan

cell therapy

Vertex Pharma’s Type 1 Diabetes Cell Therapy Shows Potential to Achieve Insulin Independence

Galapagos Says ‘No’ to SpinCo and Instead Will Seek Deals for All Assets

FDA Approves Abeona Gene Therapy for Healing Wounds From Rare, Inherited Skin Disease

CRISPR Biotech Caribou Trims Pipeline and Cuts 32% of Staff to Focus on Two Cancer Cell Therapies

Ouster of Peter Marks & Mass FDA Layoffs Have Biotech Sector Bracing for Regulatory Disruptions

Vertex Cell Therapy Misses in the Clinic, But It Has Other Shots on the Type 1 Diabetes Goal

AstraZeneca Aims to Make Cell Therapy More Accessible With $425M EsoBiotec Acquisition

Bristol Myers Squibb Buys Out Cancer Cell Therapy Partner 2seventy Bio in $286M Deal

Gene Therapy in an Implant: Neurotech Lands First FDA Approval in Rare Vision Disorder

Safety Risk Sinks Cargo Therapeutics’ Cancer Cell Therapy Prospect, Leading to 50% Staff Cut

Galapagos CEO Tells JPM Audience the Rationale for Planned Business Separation

Galapagos Keeps Focus on Cancer Cell Therapy, Spins Out Rest of Biz Into NewCo Backed by €2.4B

ASH 2024 Recap: Movement in Multiple Myeloma, Cell Therapy, Sickle Cell Disease & More

Safety Stands Out in Arcellx Cell Therapy’s ASH Data, But Don’t Overlook Manufacturing Advantages

Roche Expands Its Scope in Allogeneic Cell Therapy With $1B Poseida Therapeutics Acquisition

As Cell Therapy Gains Ground, Efforts Emerge to Improve CAR T-Manufacturing

AstraZeneca Goes With Gracell to Gain an Edge in Next-Generation Cell Therapies

Galapagos Counts On Cocoon to Help It Catch Up in Cancer Cell Therapy