Regenerative Medicine Advanced Therapy

Caribou Biosciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Monday, March 11, 2024

BERKELEY, Calif., March 11, 2024 (GLOBE NEWSWIRE) --  Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today reported financial results for the fourth quarter and full year 2023 and reviewed recent pipeline progress.

Key Points: 
  • “For our lead program, CB-010, we plan to present initial dose expansion data and the RP2D in the second quarter of 2024.
  • For our third program, CB-012, we are thrilled to have recently dosed the first patient in the AMpLify trial.
  • In December 2023, Caribou shared regulatory feedback from the U.S. Food and Drug Administration (FDA) following a Type B clinical meeting.
  • CB-012: Caribou plans to provide updates on dose escalation as the AMpLify Phase 1 clinical trial in r/r AML advances.

United States Food & Drug Administration (FDA) Supports Accelerated Approval Pathway for Rexlemestrocel-L in End-Stage Heart Failure Patients with a Left Ventricular Assist Device (LVAD)

Retrieved on: 
Sunday, March 10, 2024

“We are very pleased with FDA’s feedback that the presented results from our pivotal study of rexlemestrocel-L in end-stage HFrEF patients with LVADs may support an accelerated approval,” said Mesoblast CEO Dr. Silviu Itescu.

Key Points: 
  • “We are very pleased with FDA’s feedback that the presented results from our pivotal study of rexlemestrocel-L in end-stage HFrEF patients with LVADs may support an accelerated approval,” said Mesoblast CEO Dr. Silviu Itescu.
  • “We intend to request a pre-Biologics License Application (BLA) meeting to discuss data presentation, timing and FDA expectations for an accelerated approval filing.”
    Every year in the United States over 100,000 patients progress to end-stage HFrEF.
  • In contrast, in ischemic patients treated with rexlemestrocel-L, IL-6 levels returned to normal by 2 months and remained low through 12 months.
  • Mesoblast intends to request a pre-BLA meeting with FDA to discuss data presentation, timing and FDA expectations for an accelerated approval filing in end-stage ischemic HFrEF patients with LVAD implantation.

PRISM MarketView Highlights Work of Emerging Companies on Rare Disease Day

Retrieved on: 
Thursday, February 29, 2024

All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.

Key Points: 
  • All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.
  • Today on Rare Disease Day, PRISM MarketView highlights emerging companies working to deliver life changing treatments for those living with rare diseases.
  • RDEB is a severe rare disease characterized by extremely fragile, and extensive blistering and wounds.
  • Capricor is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases.

Mesoblast Reports Financial Results and Operational Update for Half-Year Ended December 31, 2023

Retrieved on: 
Wednesday, February 28, 2024

NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an operational update and reported financial results for the period ended December 31, 2023.

Key Points: 
  • NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an operational update and reported financial results for the period ended December 31, 2023.
  • Reduction in net cash usage for operating activities:
    For the three months ended December 31, 2023, net cash usage was US$12.3 million, a 25% reduction versus the comparative quarter in FY2023.
  • For the six months ended December 31, 2023, net cash usage was US$26.6 million, a 14% reduction versus the comparative period in FY2023.
  • Manufacturing reduced by 47% for the six months ended December 31, 2023, from US$12.8 million to US$6.7 million.

Capricor Therapeutics Announces Upcoming Type-B Meeting with the FDA to Discuss Commercial Manufacturing Planning with an Aim to Expedite BLA Pathway for CAP-1002 in Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, February 27, 2024

SAN DIEGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- – Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March.

Key Points: 
  • SAN DIEGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- – Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March.
  • In the upcoming meeting with the FDA, the Company intends to discuss its proposed chemistry, manufacturing and controls (CMC) plans for commercial launch, if approved which may enable opportunities to expedite the pathway to a Biologics License Application (BLA) submission.
  • The Company has previously been granted Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations for the use of CAP-1002 in Duchenne muscular dystrophy (DMD) and these designations enable sponsors to work closely with the FDA and receive its guidance on potential expedited pathways for approval.
  • “At Capricor, we remain focused on getting to approval as quickly as possible and we look forward to providing more details after this meeting.”

Atsena Therapeutics Receives Rare Pediatric Disease Designation from FDA for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)

Retrieved on: 
Tuesday, January 16, 2024

The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.

Key Points: 
  • The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.
  • RPD designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age.
  • “Rare Pediatric Disease designation is a significant milestone for our LCA1 program as we explore options to advance ATSN-101 into a pivotal clinical trial,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics.
  • ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment.

Caribou Biosciences Appoints Tim Kelly as Chief Technology Officer and Highlights Multiple Clinical Catalysts Expected in 2024

Retrieved on: 
Sunday, January 7, 2024

-- Caribou to present at 42nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11, at 11:15 am PST --

Key Points: 
  • In addition, Caribou highlighted successful execution across its allogeneic CAR-T cell therapy platform over the past year and provided an outlook on multiple clinical catalysts planned for 2024.
  • We are excited to kick off 2024 by welcoming Tim Kelly as Caribou’s chief technology officer,” said Rachel Haurwitz, PhD, Caribou’s president and chief executive officer.
  • Appointed Tim Kelly as Caribou’s chief technology officer, leading the company’s process development and manufacturing organizations.
  • CB-012: Caribou plans to initiate patient enrollment in the AMpLify Phase 1 clinical trial in r/r AML in H1 2024.

Caribou Biosciences Provides Regulatory Update on CB-010 Pivotal Plan with Phase 3 Trial Initiation Expected by YE 2024

Retrieved on: 
Tuesday, December 12, 2023

BERKELEY, Calif., Dec. 12, 2023 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today provided an update based on feedback from the U.S. Food and Drug Administration (FDA) following recent communications and a Type B clinical meeting regarding the CB-010 development program. The company has received the FDA’s feedback on a Phase 3 randomized controlled trial for CB-010, an allogeneic anti-CD19 CAR-T cell therapy in development for patients with second-line relapsed or refractory large B cell lymphoma (r/r LBCL). The FDA stated that Caribou’s proposed comparator arm of platinum-based immunochemotherapy followed by high dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT) is acceptable.

Key Points: 
  • The FDA stated that Caribou’s proposed comparator arm of platinum-based immunochemotherapy followed by high dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT) is acceptable.
  • “As our ongoing ANTLER trial progresses, we look forward to engaging again with the FDA ahead of initiating our planned Phase 3 pivotal trial.
  • In Q2 2024, the company plans to report initial dose expansion data from the ANTLER trial and share the recommended Phase 2 dose (RP2D) for CB-010.
  • Caribou plans to initiate a Phase 3 pivotal trial with this RP2D by year-end 2024.

Atsena Therapeutics Announces Positive 12-month Safety and Efficacy Data from Ongoing Phase I/II Clinical Trial of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)

Retrieved on: 
Monday, December 4, 2023

At 12 months post-treatment, ATSN-101 has conferred clinically meaningful improvements in vision at the highest dose with no serious treatment-emergent adverse events.

Key Points: 
  • At 12 months post-treatment, ATSN-101 has conferred clinically meaningful improvements in vision at the highest dose with no serious treatment-emergent adverse events.
  • "We continue to be encouraged by the data emerging from our Phase I/II trial of ATSN-101 in patients with GUCY2D-associated LCA1,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics.
  • “The durability of clinically meaningful visual improvements in the absence of serious treatment-related adverse events at the 12-month mark underscore the safety, tolerability, and efficacy of our subretinal gene therapy.
  • Atsena has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1.

Immatics Announces Third Quarter 2023 Financial Results and Business Update

Retrieved on: 
Tuesday, November 14, 2023

“Immatics has had a strong quarter and made steady progress both in terms of the advancement of our clinical programs and in business development.

Key Points: 
  • “Immatics has had a strong quarter and made steady progress both in terms of the advancement of our clinical programs and in business development.
  • An update on the clinical development plan is expected in the first quarter of 2024.
  • An update on the clinical development plan is expected in the first quarter of 2024.
  • On September 11, Immatics announced a strategic multi-platform collaboration with Moderna, combining Immatics’ target and TCR platforms with Moderna’s cutting-edge mRNA technology.