Cell & Gene Therapy

Uncover the transformative potential of cell and gene therapies. Delve into groundbreaking research, clinical advancements, and regulatory developments in this cutting-edge field. Stay informed about the latest breakthroughs and their impact on patient care and disease treatment.

Krystal's Vyjuvek Gene Therapy Receives FDA Approval, Boosts Sales Forecast

Krystal’s Vyjuvek Gene Therapy Gains FDA Approval, Spurs Sales Projections

Anika Sharma

Krystal Biotech’s CEO, Krish Krishnan, was quick to highlight in the company’s inaugural conference call to present its quarterly results ...

Vertex Readies for exa-cel Launch, Cautiously Eyes Enormous Opportunity

Vertex Gears Up for exa-cel Launch, Acknowledges Vast Opportunity with Cautious Outlook

Anika Sharma

After a prolonged journey in development, the first CRISPR-based gene editing therapy has recently received approval from an FDA advisory ...

Catalent Revenue at Risk After Sarepta Trial Falters

Catalent Braces for Potential Revenue Setback Following Sarepta’s Gene Therapy Trial Disappointment

Anika Sharma

Catalent, having overcome the challenges posed by the pandemic, now faces another potential setback in the form of what analysts ...

Nkarta’s cell therapy for lupus approved by FDA

Nkarta’s breakthrough cell therapy for lupus gets FDA approval after showing promise in cancer treatment

Anika Sharma

In a groundbreaking development, Nkarta is pushing the boundaries of cell therapy beyond the realm of cancer. The FDA has ...

Takeda’s Alofisel flops in phase 3 Crohn’s trial

Takeda’s Alofisel fails to meet primary endpoint in phase 3 trial for Crohn’s disease complication

Anika Sharma

Takeda, following its decision to remove an early-stage Crohn’s disease candidate from its pipeline earlier this year, is now contending ...

Kyowa Kirin to buy Orchard Therapeutics for $387M, expanding gene therapy portfolio

Kyowa Kirin to buy Orchard Therapeutics for $387M, expanding gene therapy portfolio

Anika Sharma

Kyowa Kirin has responded to the need to strengthen its late-phase pipeline by finalizing a deal to acquire Orchard Therapeutics ...

Bluebird Lonza gene therapies partnership, Zynteglo and Skysona manufacturing, Bluebird gene therapies for rare diseases, Lonza contract manufacturing services, Bluebird Zynteglo beta-thalassemia, Bluebird Skysona cerebral adrenoleukodystrophy, Bluebird Lonza collaboration agreement,

Bluebird partners with Lonza to increase production of its gene therapies for rare diseases

Anika Sharma

As bluebird bio takes flight with its high-priced gene therapies, the company is enhancing its manufacturing capabilities through a strategic ...

AbbVie drops Caribou’s CRISPR-edited CAR-T therapies after failed trials

AbbVie drops Caribou’s CRISPR-edited CAR-T therapies after failed trials

Anika Sharma

AbbVie has chosen to end its partnership with Caribou Biosciences, signaling a significant shift in its strategic focus. This decision ...

Novo Nordisk Foundation Cellerator, Cell therapy development and manufacturing, Stem cell therapies for chronic diseases, Cellerator Technical University of Denmark, Cellerator public and private clients, Cellerator funding and timeline, Cellerator cell therapy types and services,

Novo Nordisk Foundation Cellerator to boost cell therapy development and production in Denmark

Anika Sharma

The Novo Nordisk Foundation is embarking on a mission to establish a pivotal “hub” within the cell therapy ecosystem by ...

Mesoblast remestemcel-L FDA approval, Remestemcel-L cell therapy GVHD, Mesoblast Type A meeting FDA, Remestemcel-L potency assay data, Remestemcel-L single-arm trial, Mesoblast stock price MSB, Remestemcel-L pediatric and adult GVHD,

Mesoblast hopes to win FDA approval for remestemcel-L after meeting with regulators

Anika Sharma

Mesoblast, a biotech company based in Melbourne, Australia, is making determined strides toward securing FDA approval for its off-the-shelf therapy, ...

Taysha gene therapy GAN, TSHA-120 FDA feedback, Astellas Taysha gene therapy deal, Taysha pipeline update, GAN gene therapy challenges, Taysha Rett syndrome TSHA-102, Taysha stock price TSHA,

Taysha halts gene therapy for rare nerve disease after FDA and Astellas feedback

Anika Sharma

Astellas has declined the opportunity to exercise an option for an asset from Taysha Gene Therapies. Taysha, facing study design ...

Iovance Biotherapeutics, FDA, FDA delay, cell therapy, tumor-infiltrating lymphocyte, Prescription Drug User Fee Act Date, PDUFA, melanoma

Iovance’s cell therapy for skin cancer faces three-year delay due to FDA issues

Anika Sharma

The FDA has introduced a new twist in the ongoing Iovance Biotherapeutics saga, causing further delays in the approval process ...

BioMarin, Rare disease, pipeline update, Roctavian, gene therapy, hemophilia A, Pipeline addition

BioMarin R&D chief reveals two new pipeline candidates and strategy to sustain innovation

Anika Sharma

BioMarin, the California-based biotech giant, is embarking on a mission to create its most robust pipeline in history, with a ...

Calidi Biotherapeutics, SPAC, cell therapy, allogeneic, off-the-shelf treatment, Special purpose acquisition company, M&A

Calidi Biotherapeutics goes public through SPAC deal to advance off-the-shelf cancer treatments

Anika Sharma

Calidi Biotherapeutics, a San Diego-based biotech, has chosen the special purpose acquisition company (SPAC) path to enter the public market, ...

Ferring, Adstiladrin, Gene therapy, bladder cancer, FDA, early experience program

Ferring launches bladder cancer gene therapy Adstiladrin in US through early experience program

Anika Sharma

Ferring Pharmaceuticals achieved a significant milestone by administering its groundbreaking gene therapy, Adstiladrin, to its first commercial patient. This innovative ...

2seventy bio, cell therapy, Cell & Gene Therapy, layoffs, sickle cell disease, beta-thalassemia, cell therapy setback, 2seventy bio layoffs

2seventy Bio slashes 176 jobs and looks for new CEO after cell therapy setback

Anika Sharma

2seventy bio, the spinoff from bluebird bio, is undergoing significant changes under the leadership of CEO Nick Leschly. The company ...

Aphexda, a novel drug from BioLineRx, helps more myeloma patients achieve stem cell collection goals for autologous transplantation, according to a Phase 3 trial.

FDA approves Aphexda for stem cell mobilization in multiple myeloma

Anika Sharma

After a decade of limited progress, the FDA has granted approval for the use of BioLineRx’s motixafortide, also known as ...

Novartis, Gyroscope Therapeutics, restructuring, eye disease, GT005, gene therapy, geographic atrophy, ophthalmology,

Novartis drops Gyroscope’s gene therapy for eye disease after disappointing trial results

Anika Sharma

Novartis is in the process of divesting its eye disease unit, a move that includes discontinuing a geographic atrophy (GA) ...

Otsuka Pharmaceutical, psychedelics, RNA technology, adeno-associated viruses, AAV vector, ocular diseases, Shape Therapeutics

Otsuka and Shape Therapeutics join forces to create gene therapies for eye diseases using AI-powered platform

Anika Sharma

Just a week after Otsuka Pharmaceutical ventured into the psychedelics sector, the Japanese pharmaceutical giant has struck a collaboration deal ...

cell therapy manufacturing, funding round, startups, Cell & Gene Therapy

BlueWhale Bio Emerges With $18m To Tackle Cell Therapy Manufacturing Challenges

Anika Sharma

BlueWhale Bio, a spinout from the University of Pennsylvania, has emerged with a bold mission to tackle bottlenecks in the ...

Kriya Therapeutics, NASH, Cell & Gene Therapy, M&A, nonalcoholic steatohepatitis

Kriya Acquires Gene Therapy Biotech To Enter NASH Market With Novel Approach

Anika Sharma

Kriya Therapeutics, a prominent player in the biotech industry, has continued its acquisition spree, this time securing Tramontane Therapeutics to ...

Mesoblast Cell and Gene Therapy, remestemcel-L, steroid-refractory acute graft versus host disease

Mesoblast CEO Slashes Salary by 30% to Fund Cell Therapy Trial

Anika Sharma

In a bold move reflecting commitment to the company’s mission, Mesoblast’s CEO has chosen to lead by example by implementing ...

Bristol Myers Squibb, Cellares, CAR-T Therapy, Blood cancer, Abecma, Breyanzi, cell and gene therapy

Bristol Myers Squibb’s Bold Robotic Alliance with Cellares for Advanced CAR-T Production

Anika Sharma

In the realm of cutting-edge cell therapy, where innovation is the heartbeat and the promise of transformative treatments is the ...

Bayer, Cell Therapy, Parkinson’s Disease, Clinical trial results, Parkinson Cell Therapy,

Bayer Advances Parkinson’s Cell Therapy to Phase 2 Trial

Anika Sharma

A new horizon gleams for Bayer’s quest to conquer Parkinson’s disease through advanced cell therapy. In a significant revelation, the ...

Ferring Pharmaceuticals, Royalty Pharma, nadofaragene firadenovec, Bladder cancer, non-muscle invasive bladder cancer, Adstiladrin

Ferring Strikes $500M Agreement with Royalty Pharma for Innovative Bladder Cancer Gene Therapy

Anika Sharma

Royalty Pharma has carved a distinctive niche in the pharmaceutical landscape, specializing in identifying innovative products both pre- and post-approval, ...

Fulcrum Therapeutics, Sickle cell disease, Clinical hold, FDX-6058

Fulcrum’s Sickle Cell Disease Program Resumes as FDA Lifts 6-Month Hold

Anika Sharma

Fulcrum Therapeutics’ ambitions in the realm of sickle cell disease (SCD) have been revitalised after a six-month hiatus. The FDA ...

Biotechnology, Cell and gene therapy, Nobel Prize, Special purpose acquisition company, SPAC

Nobel Prize Winner’s Biotech Company Escapes the Graveyard with SPAC

Anika Sharma

Reviving a Silent Spell: Celixir, a UK-based cell therapy biotech, emerges from the shadows with a transformative move. Following a ...

Henrietta Lacks’ Family Sues Ultragenyx for Gene Theft

Gene Therapy Vector Lawsuit: Henrietta Lacks’ Estate Targets Ultragenyx Over Exploitation

SG Tylor

Just under a month after reaching a settlement with Thermo Fisher over the utilization of Henrietta Lacks’ cells, her family ...

NICE Rejects CSL’s Hemophilia B Gene Therapy

Despite CSL’s hemophilia B gene therapy, NICE is not persuaded

SG Tylor

The UK’s National Institute for Health and Care Excellence (NICE) has expressed hesitation in recommending approval for CSL Behring and ...

Sensorion Seeks Approval for Clinical Trial of OTOF-GT Gene Therapy Candidate in the UK

Sensorion Seeks Approval for Clinical Trial of OTOF-GT Gene Therapy Candidate in the UK

SG Tylor

Source – Sensorion On July 10, Sensorion filed its first regulatory application to start human testing of OTOF-GT, a gene ...

12 Next