Concha bullosa

Abeona Therapeutics Reports Full Year 2023 Financial Results and Announces Completion of FDA Inspections

Retrieved on: 
Monday, March 18, 2024
Research, AAV, Advisory Committee, Form FDA 483, PLI, Sale, EIR, University, Trial of the century, Rett syndrome, Concha bullosa, Priority review, Investment, IP, Therapy, Safety, REMS, EB, Food, FDA, BLA, RDEB, PDUFA, Pharmaceutical industry

On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.

Key Points: 
  • On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.
  • In January 2024, the FDA completed both a Bioresearch Monitoring (BIMO) inspection of Abeona and the Mid-Cycle Meeting regarding the pz-cel BLA.
  • Following completion of the BIMO inspection, the FDA inspector did not issue any observations or FDA Form 483s.
  • In March 2024, Abeona received a written Establishment Inspection Report (EIR), the formal report from the FDA regarding the BIMO inspection.

Global Cell Counting Market to Hit $15.72 Billion by 2029, Fueled by Advancements in Cell Biology and Therapy - ResearchAndMarkets.com

Retrieved on: 
Friday, March 29, 2024
Research, Technology, University, Genetics, Artificial Intelligence, Education, Science, Nanotechnology, Biotechnology, Stem Cells, Other Technology, Health, Other Science, Automation, Quality control, Interdisciplinarity, Acute lymphoblastic leukemia, Growth, Prevalence, Application, Novartis, Patient, Conditional sentence, Cell biology, Lung, Concha bullosa, Hospital, Artificial intelligence, National Cancer Institute, AI, Regenerative medicine, Duchenne muscular dystrophy, Therapy, DEB, Miniaturization, Sarepta Therapeutics, CGT, FDA, DMD, Tisagenlecleucel, PDUFA, Pharmaceutical industry

The global cell counting market was valued at US$9.98 billion in 2023 and is expected to reach US$15.72 billion by 2029.

Key Points: 
  • The global cell counting market was valued at US$9.98 billion in 2023 and is expected to reach US$15.72 billion by 2029.
  • Advancements in cell biology, biotechnology, and medical research are driving demand for precise cell counting, especially with the rise of personalized medicine and regenerative therapies.
  • PDUFA dates for therapies could translate into an order book boost for the cell counting players in the market, resulting in market growth.
  • The global cell counting market is consolidated, with top five players holding approximately 70% share of the market.

PRISM MarketView Highlights Work of Emerging Companies on Rare Disease Day

Retrieved on: 
Thursday, February 29, 2024
Rare disease, Therapy, Regenerative Medicine Advanced Therapy, FAP, Concha bullosa, Sulindac, Chemistry, Fast track (FDA), Muscular dystrophy, FDA, Sensation, News, Eflornithine, DMD, Prader–Willi syndrome, BLA, Chromosome, RDEB, PDUFA, Skin, Voyager, PRISM, Duchenne muscular dystrophy, Safety, Neurocrine Biosciences, Rare Disease Day, Genetics, Fatigue, Walking, PWS, Patient, DSM-IV codes, Medicine, Disease, Exosome, FA, CMC, Priority review, Pharmaceutical industry

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.

Krystal Biotech Announces Publication in the New England Journal of Medicine on the Application of B-VEC to Treat Ocular Complications in Patient with Dystrophic Epidermolysis Bullosa

Retrieved on: 
Thursday, February 8, 2024
Manuscript, KRYS, Paratriathlon, Pannus, Patient, Disease management, Scar, Ophthalmology, FDA, Abrasion, Concha bullosa, DEB, Risk, Research and development, Gene, Blister, Associate, Suma, Doctor of Philosophy, NEJM, Eye, Pharmaceutical industry

PITTSBURGH, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced data on the compassionate use of beremagene geperpavec (B-VEC), administered as an eyedrop to treat a patient with dystrophic epidermolysis bullosa (DEB) with cicatrizing conjunctivitis has been published in the New England Journal of Medicine (NEJM). The full manuscript, titled “Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa,” can be found in the February 8, 2024 issue of the NEJM.

Key Points: 
  • The full manuscript, titled “Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa,” can be found in the February 8, 2024 issue of the NEJM.
  • Over 25% of patients with DEB develop ocular complications such as corneal erosions, abrasions, blistering and scarring that can lead to impaired vision.
  • The NEJM publication describes the first application of B-VEC to treat ocular complications in a patient with DEB under a compassionate use program.
  • If approved, this approach could drastically benefit these patients.”
    A patient presented with severe cicatrizing conjunctivitis secondary to DEB.

Abeona Therapeutics Announces Progress Update on Pz-cel Biologics License Application (BLA)

Retrieved on: 
Thursday, February 1, 2024
REMS, Research, Patient, RDEB, FDA, Marketing, Advisory Committee, Biologics license application, Food, PDUFA, Concha bullosa, BLA, Medical device

CLEVELAND, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced, as part of the review process by the U.S. Food and Drug Administration (FDA) for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB), completion by the FDA of both a Bioresearch Monitoring (BIMO) inspection of Abeona and the BLA mid-cycle review meeting.

Key Points: 
  • The FDA inspector did not issue any observations or FDA Form 483s during the inspection.
  • The formal report from the FDA regarding the BIMO inspection will be received at a later date.
  • Following the BIMO inspection, the BLA mid-cycle review meeting took place on January 25, 2024.
  • The FDA reaffirmed its earlier indication that it does not currently plan to convene an Advisory Committee for pz-cel.

Abeona Therapeutics Announces $50 Million Credit Facility

Retrieved on: 
Monday, January 8, 2024
Marketing, Food, Concha bullosa, Priority review, FDA, PDUFA, PZ, RDEB, Biologics license application, Patient, COL7A1

CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.

Key Points: 
  • CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.
  • The credit agreement, which has a term of three and a half years, includes a first tranche of $20 million at closing, a second tranche of $10 million of committed capital, and an additional accordion option to upsize the credit facility by an additional $20 million upon satisfaction of certain terms and conditions.
  • The FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel.
  • “We are excited to enter into this relationship with Avenue Venture Fund and secure additional financial resources to further support our launch and commercialization efforts for pz-cel,” said Joe Vazzano, Chief Financial Officer of Abeona.

Krystal Biotech Receives Orphan Drug Designation from the Japanese MHLW for Beremagene Geperpavec-svdt (B-VEC)

Retrieved on: 
Tuesday, December 19, 2023
Research and development, Suma, Concha bullosa, Research, MHLW, ODD, DEB, KRYS, Patient, Safety, Pharmaceutical industry

PITTSBURGH, Dec. 19, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, today announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation (ODD) to beremagene geperpavec-svdt (B-VEC or VYJUVEK) for the treatment of dystrophic epidermolysis bullosa (DEB).

Key Points: 
  • PITTSBURGH, Dec. 19, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, today announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation (ODD) to beremagene geperpavec-svdt (B-VEC or VYJUVEK) for the treatment of dystrophic epidermolysis bullosa (DEB).
  • “Receiving ODD is an important step in expediting the development of B-VEC in Japan and underscores the unmet needs of patients with DEB,” said Suma Krishnan, President, Research & Development, Krystal Biotech, Inc. “We look forward to working closely with MHLW to bring this important treatment to patients in Japan as soon as possible.”
    The orphan drug designation system in Japan aims to support the development of drugs for diseases that affect fewer than 50,000 patients in Japan, for which significant unmet medical need exists.
  • An investigational therapy is eligible to qualify for ODD if there is no approved alternative treatment option or if there is high efficacy or safety compared to existing treatment options expected.
  • Specific measures to support the development of orphan drugs include subsidies for research and development expenditures, prioritized consultation regarding clinical development, reduced consultation fees, tax incentives, priority review of applications, reduced application fees, and extended registration validity period.

Krystal Biotech Announces EMA Validation of Marketing Authorization Application for VYJUVEK for the Treatment of Dystrophic Epidermolysis Bullosa

Retrieved on: 
Monday, November 27, 2023
Research and development, Marketing, Suma, Concha bullosa, EMA, MAA, Orphan, DEB, KRYS, Committee, Patient, CHMP

PITTSBURGH, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, today announced that the Company’s Marketing Authorization Application (MAA) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for VYJUVEK (beremagene geperpavec-svdt, also known as B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB) has been validated and is now under CHMP review.

Key Points: 
  • PITTSBURGH, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, today announced that the Company’s Marketing Authorization Application (MAA) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for VYJUVEK (beremagene geperpavec-svdt, also known as B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB) has been validated and is now under CHMP review.
  • A CHMP opinion is anticipated in the second half of 2024.
  • Based on this positive opinion, the Company would be eligible for up to an additional two years of marketing exclusivity in the EU, on top of the ten-year EU market exclusivity after market approval in the EU.
  • Previously, VYJUVEK received Orphan Drug Designation and PRIority Medicines (PRIME) eligibility from the EMA.

Abeona Therapeutics Announces FDA Accepts and Grants Priority Review for Pz-cel Biologics License Application (BLA)

Retrieved on: 
Monday, November 27, 2023
Quality of life, Priority review, Food, Concha bullosa, ISID, BLA, FDA, PDUFA, Biologics license application, Advisory Committee, Patient, Safety, Wound healing, COL7A1, Pharmaceutical industry

CLEVELAND, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024. The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application.

Key Points: 
  • Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024.
  • The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application.
  • Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction.
  • The grant of the Priority Review status is an important prerequisite for Abeona’s eligibility for a Priority Review Voucher.

Abeona Therapeutics Reports Third Quarter 2023 Financial Results and Corporate Developments

Retrieved on: 
Monday, November 13, 2023
World Health Organization, Priority review, Bristol Myers Squibb, Safety, Business development, Food, Therapy, BLA, International, Investment, COL7A1, Research, FDA, EB, BMS, Indiana University, Concha bullosa, PZ, Pharmaceutical industry, Cryptocurrency, RDEB

CLEVELAND, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the third quarter of 2023 and provided corporate updates.

Key Points: 
  • The FDA also agreed that retroviral vector manufactured at Abeona and Indiana University appear comparable based on the data that Abeona provided in its briefing book.
  • If accepted with Priority Review, Abeona expects potential BLA approval in the second quarter of 2024.
  • Abeona estimates that its cash and cash equivalents, restricted cash and short-term investments as of September 30, 2023 are sufficient resources to fund operations into the fourth quarter of 2024.
  • Abeona Therapeutics will host a conference call and webcast today, November 13, 2023, at 8:30 a.m.