Abstract
Regulatory Wire
EMA Celebrates 10 Year Anniversary of Orphan Drug Legislation
The European Medicines Agency (EMA) held a two day conference commemorating the 10th anniversary of the passage of Orphan Drug Regulation by the European Union (EU). This legislation was brought forward in order to improve the offer of products for patients with rare conditions by providing drug developers with a bevy of economic and regulatory incentives, including 10-year market exclusivity, protocol assistance, and access to the Centralised Procedure for Marketing Authorisation. The two day EMA conference, whose aim was to review the success and future prospects of the EU's Orphan Drug law, was attended by representatives from the European Parliament, the European Commission, international and European regulatory agencies, members of the Committee for Orphan Medicinal Products (COMP), patient groups, health professionals, and pharmaceutical industry to review the impact of ten years of orphan medicines legislation and to look ahead at future opportunities and challenges. Since being signed into law in April 2000, EMA has received more than 1100 applications, of which 720 diseases have been granted orphan designation. A total of 62 orphan designated medicines for 53 different rare diseases have been approved for use in the EU. (sk)
International Stem Cell Workshop Provides Guidance for Clinical Trials
Last month, the EMA held a workshop to review the opportunities and difficulties in developing stem cell-based therapies and to discuss regulatory challenges. According to the EMA, this unique workshop has “assembled for the first time European and international experts from academia, regulatory authorities (from Europe, Japan and the United States) and pharmaceutical industry.” Currently within the European Union, there are some 40 clinical trials being conducted exploring the use of stem cells in regeneration of lost or damaged tissue (heart, skin, bone, spinal cord, liver, pancreas and cornea) and in haematological or solid-organ malignancies. The majority of these trials are using mesenchymal cells derived from adipose tissue, bone marrow, stromal cells and connective tissue. A small proportion of the trials are using haematopoietic stem cells. Presentations of the keynote speakers at this workshop can be found here:
NIH Safety Symposium Examines Gene Modified T Cells
On June 15, 2010 the NIH's recombinant DNA advisory committee (RAC) held a one day public safety symposium entitled “Gene-Modified T Cells: Challenges in Clinical Trial Design”. This meeting brought together leading experts in the field of gene modified T cells from clinicians to scientists to members of industry and government. Among the issues covered was an examination of “Serious Adverse Events in Trials Utilizing Chimeric Antigen Receptors: Lessons for Trial Design” and “Points to Consider for Trial Design Involving New Receptors: Initial Dose, Co-signaling Moieties, and Subject Selection”. Full details of the symposium can be found at:
Top Ten Biotech/Pharmaceutical Patent Claimants of 2009
The Intellectual Property Owners Association (IPO), a trade association for owners of patents, trademarks, copyrights and trade secrets, released its 27th annual list of the top 300 organizations receiving U.S. patents in 2009. Of the top 300 organizations, 47 were biotech and pharmaceutical companies. The top ten biotech/pharmaceutical companies that received patents last year were: Du Pont (585 patents) Johnson and Johnson (487 patents) Medtronic (421 patents) Industrial Technology Research Institute (421 patents) Merck (340 patents) Pfizer (280 patents) Monsanto (259 patents) Hoffman-La Roche (257 patents) Reagents of the University of California (251 patents) Boston Scientific Corporation (250 patents)
Science Wire
AAV Needs AAP
The single-stranded DNA genome of adeno-associated virus (AAV) is encased in a 20–25 nm diameter, icosahedral protein capsid. This capsid is composed of 60 protein subunits, designated VP1, VP2, and VP3, that are arranged in a T = 1 icosahedral symmetry. Alternative splicing and translation initiation of VP2 at a nonconventional ACG initiation codon promote the expression of VP1, VP2, and VP3 in a stoichiometric ratio of 1:1:10. The diversity amongst different AAV isolates has been primarily traced to the major capsid protein (VP3); differences in the amino acid sequence of the VP3 subunit of different AAV isolates determine capsid surface topology, which leads to the observed diversity in receptor usage, antigenicity, tissue tropism, and host specificity of AAV variants and serotypes. It has been suggested that VP3 undergoes self-assembly to form icosahedral virion shells. Now, the group of Dr. Jürgen A. Kleinschmidt at the German Cancer Research Center in Heidelberg, Germany reveals a new player in AAV capsid assembly.
Using trans-complementation assays, the authors showed that a protein encoded by open reading frame (ORF) 2 of the AAV2 cap gene provides vital capsid assembly helper function. Sequence analysis revealed that this ORF is translated out of frame with respect to VP3. The authors went on to demonstrate that this novel protein, called assembly-activating protein (AAP), is 23 kDa in size and that it stimulates the transport of unassembled VP proteins into the nucleolus for capsid assembly. Importantly, using sequence analysis, the authors suggest that all other species of the genus Dependovirus encode a protein homologous to AAP in their cap gene. (sk)
Industry Wire
