CGT Milestones 4/6

Illustration of a biotech research team working on gene therapy for inherited eye disease in a clean modern lab setting in Europe

Barcelona-Based SpliceBio Raises €119M to Target Stargardt Disease

Published on Spencer Knight via LinkedIn SpliceBio has secured €119 million in Series B funding, the largest ever for a biotech in...

CGT Milestones

Jun 17

Photo of a smiling mother recovering in a hospital room with medical staff nearby, symbolizing recovery from advanced melanoma using TIL therapy

Mother of Two Enters Remission After TIL Cell Therapy for Stage 4 Melanoma

Published on Spencer Knight via LinkedIn Missy Christy had already beaten cancer once. Then, as a new mother, she faced it again—this...

CGT Milestones

Jun 17

Concept image of dual-target CAR-T therapy and dendritic cell treatment in a clinical setting, representing progress in glioblastoma cell therapy research

CAR-T and Dendritic Cell Therapies Show Promise in Tackling Glioblastoma

Published on Spencer Knight via LinkedIn Glioblastoma remains one of the deadliest brain cancers with limited treatment options. Two new...

CGT Milestones

Jun 17

Concept image of two pharmaceutical companies merging over molecular designs, symbolizing a strategic immunology acquisition by Sanofi

Sanofi Acquires Blueprint Medicines for $9.1B, Strengthens Rare Immunology Portfolio

Published on Spencer Knight via LinkedIn Sanofi has announced a definitive agreement to acquire Blueprint Medicines in a $9.1 billion...

CGT Milestones

Jun 17

Image of a young woman smiling and active outdoors after recovering from lupus with CAR T-cell therapy, symbolizing restored mobility and remission

Australian CAR T Trial Puts Lupus Into Remission for Young Woman in Historic First

Published on Spencer Knight via LinkedIn Lani Watson, 32, lived with severe lupus that left her in constant pain and unable to walk. In a...

CGT Milestones

Jun 17

Concept image of a business handshake over a 3D brain model, representing a neuroscience acquisition focused on Alzheimer’s and microglial therapy strategy

Sanofi Acquires Vigil Neuroscience for $470M, Expands Alzheimer’s Pipeline

Published on Spencer Knight via LinkedIn Sanofi has agreed to acquire Vigil Neuroscience in a $470 million deal, reinforcing its...

CGT Milestones

Jun 17

https://www.linkedin.com/posts/spencer-knight_genetherapy-cellandgenetherapy-biotech-activity-7330941740536782850-sbMB/?utm_source=share&utm_medium=member_ios&rcm=ACoAAEHdwkIBMc0_k-QYY8yDdD5xx4gDWJ-c8bM

Gene Therapy Frees Patient from 29 Years of Haemophilia B Treatment

Published on Spencer Knight via LinkedIn Elliott Collins, 34, lived with haemophilia B for nearly three decades, relying on frequent...

CGT Milestones

Jun 17

Illustration of siRNA molecules silencing genes inside cells, symbolizing therapeutic breakthroughs in RNA-based treatments across multiple biotech partnerships

2025 Set to Be a Breakout Year for siRNA as Funding and FDA Wins Pile Up

Published on Spencer Knight via LinkedIn siRNA therapies are seeing renewed momentum, with major investments and regulatory wins...

CGT Milestones

Jun 17

Illustration of a baby in a hospital crib surrounded by clinicians and CRISPR research visuals, symbolizing personalized gene-editing therapy for a rare condition

Baby KJ Becomes First Patient to Receive Fully Personalized CRISPR Therapy

Published on Spencer Knight via LinkedIn At just nine months old, KJ was diagnosed with CPS1 deficiency, a rare metabolic disorder...

CGT Milestones

Jun 17

Illustration of biotech firms advancing cell and gene therapy through funding rounds, approvals, and RMAT designations in a dynamic clinical and regulatory landscape

$240M+ in Deals and 1 New Approval Mark CGT Momentum Surge at ASGCT

Published on Spencer Knight via LinkedIn A packed week at ASGCT brought a wave of new CGT developments totaling over $240 million in...

CGT Milestones

Jun 17

Illustration of pharmaceutical collaboration focused on liver disease therapy, showing a symbolic liver and molecular targets representing GSK’s acquisition deal

GSK Acquires Efruxifermin for $2B, Targeting MASH and Liver Fibrosis

Published on Spencer Knight via LinkedIn GSK is acquiring efruxifermin alfa from Boston Pharmaceuticals for up to $2 billion, including...

CGT Milestones

Jun 17

Illustration of a newborn receiving supportive care for brain injury, symbolizing regenerative therapy for hypoxic birth-related conditions in a neonatal unit

HAON Life Sciences Secures €10.7M to Advance Allogeneic Cell Therapy for Infant Brain Injury

Published on Spencer Knight via LinkedIn HAON Life Sciences, a Dublin-based biotech startup, has received €10.7 million from Ireland’s...

CGT Milestones

Jun 17

Portrait of a young woman smiling during recovery in a hospital room after CRISPR therapy for late-stage colon cancer

CRISPR Eliminates Stage 4 Colon Cancer in Breakthrough First-in-Human Trial

Published on Spencer Knight via LinkedIn Emma Dimey was diagnosed with stage 4 colorectal cancer at just 23. After 11 years of failed...

CGT Milestones

Jun 17

Portrait of a young boy in a hospital bed recovering from gene therapy for a rare immune condition, smiling brightly with supportive surroundings

Gene Therapy Saves 4-Year-Old with Rare Immune Disorder Once Considered Fatal

Published on Spencer Knight via LinkedIn Eisa Hussain was diagnosed as an infant with leukocyte adhesion deficiency type 1 (LAD-1), a...

CGT Milestones

Jun 17

Concept image of a biotech acquisition with RNA molecular symbols and kidney treatment themes representing Novartis and Regulus deal

Novartis to Acquire Regulus for $1.7B, Advancing MicroRNA Therapies

Published on Spencer Knight via LinkedIn Novartis is acquiring Regulus Therapeutics for up to $1.7 billion to expand its position in...

CGT Milestones

Jun 17

Illustration of a pediatric patient receiving a gene-corrected skin graft for a rare genetic skin condition in a modern clinical setting

Abeona’s ZEVASKYN™ Becomes First Cell-Based Gene Therapy Approved for RDEB

Published on Spencer Knight via LinkedIn The FDA has approved ZEVASKYN™, the first-ever autologous cell-based gene therapy for patients...

CGT Milestones

Jun 17

Image of a clean and modern biotech facility on a sunny day, symbolizing successful commercialization of gene therapy products

Ferring’s ADSTILADRIN Hits €70M in Year One, Validating Gene Therapy Market Growth

Published on Spencer Knight via LinkedIn Ferring Pharmaceuticals has emerged as a commercial frontrunner in gene therapy with the...

CGT Milestones

Jun 17

Portrait of a smiling woman in her late 50s at home with natural light, symbolizing recovery and renewed life after CAR-T therapy for lymphoma

From 2 Weeks to Cancer-Free: Lisa Noble’s Remarkable CAR-T Therapy Outcome

Published on Spencer Knight via LinkedIn Lisa Noble, 58, was given just two weeks to live after failing chemotherapy for B cell lymphoma....

CGT Milestones

Jun 17

Photo of a clinical researcher preparing a cancer vaccine in a sterile lab setting as part of a melanoma immunotherapy trial in the UK

10,000 Cancer Patients to Gain Fast-Track Access Through NHS Melanoma Vaccine Trial

Published on Spencer Knight via LinkedIn The NHS in England is fast-tracking a cancer vaccine trial for melanoma through its Cancer...

CGT Milestones

Jun 17

Illustration of a lipid nanoparticle delivering CAR mRNA to T cells for in vivo therapy, set in a clean lab environment with global clinical trial icons

Capstan Launches In Vivo CAR-T Trial Using LNP Technology for Oncology

Published on Spencer Knight via LinkedIn Capstan Therapeutics has entered Phase 1 trials with CPTX2309, an in vivo CAR-T candidate that...

CGT Milestones

Jun 17

Barcelona-Based SpliceBio Raises €119M to Target Stargardt Disease

Mother of Two Enters Remission After TIL Cell Therapy for Stage 4 Melanoma

CAR-T and Dendritic Cell Therapies Show Promise in Tackling Glioblastoma

Sanofi Acquires Blueprint Medicines for $9.1B, Strengthens Rare Immunology Portfolio

Australian CAR T Trial Puts Lupus Into Remission for Young Woman in Historic First

Sanofi Acquires Vigil Neuroscience for $470M, Expands Alzheimer’s Pipeline

Gene Therapy Frees Patient from 29 Years of Haemophilia B Treatment

2025 Set to Be a Breakout Year for siRNA as Funding and FDA Wins Pile Up

Baby KJ Becomes First Patient to Receive Fully Personalized CRISPR Therapy

$240M+ in Deals and 1 New Approval Mark CGT Momentum Surge at ASGCT

GSK Acquires Efruxifermin for $2B, Targeting MASH and Liver Fibrosis

HAON Life Sciences Secures €10.7M to Advance Allogeneic Cell Therapy for Infant Brain Injury

CRISPR Eliminates Stage 4 Colon Cancer in Breakthrough First-in-Human Trial

Gene Therapy Saves 4-Year-Old with Rare Immune Disorder Once Considered Fatal

Novartis to Acquire Regulus for $1.7B, Advancing MicroRNA Therapies

Abeona’s ZEVASKYN™ Becomes First Cell-Based Gene Therapy Approved for RDEB

Ferring’s ADSTILADRIN Hits €70M in Year One, Validating Gene Therapy Market Growth

From 2 Weeks to Cancer-Free: Lisa Noble’s Remarkable CAR-T Therapy Outcome

10,000 Cancer Patients to Gain Fast-Track Access Through NHS Melanoma Vaccine Trial

Capstan Launches In Vivo CAR-T Trial Using LNP Technology for Oncology

Sarawak Society for Cell & Gene Therapy Research

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